Dr. Sheerene Idriss explains one of the biggest factors thats aging you that you may never have heard of before.

As the saying goes, new year, new you, right? It depends on who youre speaking with, but if youre asking your dermatologist, they might explain that its not that simple. Theres no magical serum or cream that can completely turn back the hands of time, but with the recent innovations in cosmetic procedures, it is easier than ever to help your skin age gracefully. Fillers especially are one of the most popular options for people who are looking to lift, firm, or otherwise minimize obvious signs of aging, like fine lines and wrinkles.

There are always new research and innovations within the world of dermatology, so we sat down with top NYC dermatologist Dr. Sheerene Idriss to get the 411 on the most exciting breakthroughs for 2020. From the two new filler formulations to the most requested cosmetic proceduresplus one of the biggest factors thats aging you that you might never have heard of beforethis is what we can look forward to in the skin-care market in the year ahead.

What has been the most requested procedure in the past few months, and what do you expect to continue throughout 2020?

A lot of jawline addressment, wherein people want a more rigid, firmer, or more defined jawline. Not a different-looking jaw, which I have to re-educate some people aboutyou have to work in sync with the face. A strong jawline definitely saves the face as we age, since our chins and our jawlines sag. But your jawline has to align with your mid-face. It has to co-exist in a way that makes sense.

Why do you think that is becoming such a popular request?

I think its Instagram. Its both a blessing and a curse. People are oftentimes seeing versions of themselves that are maybe not real. But even if you think of the big-name celebrities, like Kim Kardashian or Jennifer Lopez, they have very strong jawlines. They are associating it with being beautiful. Some patients are coming in with that mind-set, versus being an actual candidate for that procedure. Its really up to the physician to be true to themselves so that people dont start morphing into these caricatures were seeing. [Some of] these crazy jawline fillers almost look like theyve gotten an implant. It really doesnt match the face.

What about in terms of filler innovations?

There was a rumor about Velitte [coming] out in 2020. Itll be interesting to see how a lightweight hyaluronic filler can replenish moisture in the dermis. [Its] a lighter effect that helps with the texture of the skin. I also heard that Revance, the approved form of Teoxanewhich is a resilient form of hyaluronic acid, meaning its longer-lastingitll be interesting to see how well incorporate it when using various fillers in the face. Theyve been using it outside of the United States for a while now. With regards to Teoxane, they have four different ones, three of which have been FDA-approved in the US, and theyre all indicated for different areas of the face. The fourth one is still going through approval. Its like having different paintbrushes in your toolbox.

Is there anything that doesnt yet exist that youre hoping to see developed within the world of fillers?

I think that facial anatomy is so complicated when it comes to structure. There is no miracle product, like in skin care. I dont think it would be good to have a filler that could address all of your problems, because when you think of the face, you want everything to work a little differently. You want it to be sturdier around the jawline to hold the face up, or along your cheekbones. You want it to be malleable along the cheeks or where you smile, so your face can move. It doesnt make sense to have a miracle filler, in my mind.

What about longer-lasting filler?

It makes sense to me to have a long-lasting filler; however, it comes with a caveat. I dont think that having super long-lasting filler is really beneficial because our bone structure changes so much as we age, especially in our 40s. So if you have a long-lasting filler over an ever-changing face, things can start to look wonky over time.

If you look at [cheek] implants, you would have these patients come in, and there are a few who are a bit older because its not as trendy now or en vogue, but their cheeks are sitting [higher] and everything is aging around it, [so] they have a Cruella de Vil look because its not moving. With an aging face, you have to be malleable; you have to work with an aging face. You cant be rigid.

Illustration: Meghann Stephenson... Read More

How much bone density are you losing when you start to age? What is the average percentage per decade?

It starts in your 20s. There is a study from 2012 [stating that] bone structure reduces about 90 percent after menopause. Aging is associated with the decrease in the growth hormone secreted in the pituitary gland. Its decreasing at 14 percent per decade. I dont know if this is associated with bone resorption, but its significant. It cant be overlooked. [Which is why] I feel strongly about not using semi-permanent fillers in the face. I think you can use them in different areas of the face to your benefit, but in the midface, area thats actively changing, you have to grow with the face.

Along with jaw filler, what is another type of treatment that youre seeing more often with your patients?

Younger and younger women are asking for under-eye [filler]. I think it has to do with the filter effect [thats] going on, where people think they shouldnt have a little line under their eyes when they smile. Id say thats normal. Sometimes you definitely should [explore under-eye filler] if the wrinkle is a little deeper, but Id say those two [are the most popular].

What is the process for using the filler under the eye?

Its a tricky area. I think more people think they need it, that theyve lost volume in the eye area, when really their face is dropping. Its better to lift the face laterally with filler around the eye, in my opinion, without really going for the under-eye filler. There are two ways you can do filler: with a blunt-edge needlea cannulaor a sharp-edge needle. But it really depends on what the physician is comfortable using.

Always [use] a lightweight filler for under the eye. I personally dont do the eye without addressing the rest of the face. If youre scared of fillers, you can always do the PRP [platelet-rich plasma] injections, which take your own blood and separate the growth factors. Its still an invasive procedure, but its coming from yourself.

As fillers have become more socially acceptable, do you think people are shying away from more invasive procedures, or are they fatigued by fillers and are searching for something else long-term?

Im very biased because I only see my patient population. The first question I always ask an older patient is Are you someone who wants to get a face-lift in this lifetime, or are you completely against it? The person who is OK with it, Im looking at their skin quality and anatomy and seeing if theyre a candidate for a face-lift. I would push them to get one, and then we can maintain it when they come back to me. A lot of people ask me to reverse or fix work. I think, unfortunately, there are a lot of people out there who just listen to exactly what their patient wants, and that can result in unnecessary amounts of filler to the face, without thinking about the surgical procedure as a choice.

I always think about whether or not this age [at which to recommend a face-lift] will be pushed back because of all of the advances weve made to tighten and resurface the skin. I think that the age of having to get a face-lift is going to be pushed back over time. [Right now] people start to take it seriously at 55-plus.

What is the average age of the first-time filler user in your practice?

Ive noticed three main peaks. The first one is 30 to 33, where they feel like over the past six months their face has shifted. The second one is 39 to 42, where the change happens more dramatically, maybe over a month or so. And then again, in your late 40s, where I get told over and over again, I woke up one morning and I dont recognize myself. Help. Volume loss starts to happen in your late 20s, early 30s, which makes sense if youre thinking about bone density and all of that. Youve probably lost weight, gained weight, et cetera.

For a client whos coming in and saying I dont understand why I look older, but I do. Please help. What is your strategy?

I really look at the face. When youre looking at aging, its a number of things. Its volume loss; its if you have any built-in wrinkles in your face; its the color of your skin; its how elastic your skin is, and your bone structure. I will start with one and see if it works. Some people want Botox but have no lines. I try to tell them, You dont need it yet. There are women out there that listened to their moms [to take care of their skin] and look like little fairies and look impeccable. But theres always the one beginning sign of [aging].

Photo: Shot on site at 6 Columbus, a Sixty Hotel. On Jonelle: Necklace, Baker & Black,Catbird; Hair, Angela Soto; Makeup, Andriani.

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Five-year-old Emlee jumps high, runs fast and likes to pirouette around the living room in her white ballet slippers.

Her mom, Karalayne Maglinte, calls her a miracle. Indeed, Emlee is the embodiment of the word: Shes one of the reasons Maglinte is alive today. Another reason: High-risk pregnancy physicians and cancer specialists at UCI Health were able to help the Fontana woman when no one else could.

Cancer during pregnancy is a rare event, occurring approximately once per 1,000 pregnancies annually, according to the National Institutes of Healths World Journal of Oncology. Fortunately, we have plenty of experience treating patients who need a multidisciplinary approach, says Dr. Rita Mehta, a UCI Health oncologist.

Mehta has cared for several pregnant women with cancer, including Michelle Clark-Salib, who was just 28 when diagnosed with breast cancer. Her son Caleb is now 7, and Clark-Salib is cancer-free.

But the situation was touch-and-go for a long time, just as it was for Karalayne and Emlee Maglinte. Their poignant story began to unfold in 2013 when Maglinte was 15 weeks pregnant. She was 36 years old and had two boys at home: Ian, 6, and Isaac, 18 months.

Because I was pregnant, I was much more aware of my bodys cues that something wasnt right, and I was quicker to react than I might have been otherwise, Maglinte says.

My hands and feet were itchy, she says, so itchy I wanted to tear them off. She consulted Dr. Google and read that it might be a liver issue.

At first I thought perhaps it was because I was pregnant with a girl, and the other two were boys, Maglinte recalls. But it got so intense that I began to worry. I didnt want to endanger her. The itchiness began on a Friday. By Monday, she was convinced she needed to call her doctor.

An arduous round of tests, physician appointments and hospitalizations ensued as several Inland Empire doctors tried unsuccessfully to diagnose and treat Maglinte. In addition to the itchiness, she developed jaundice. After four days at a community hospital, she was taken by ambulance in the middle of the night to UCI Douglas Hospital, in Orange.

A team quickly assembled, including high-risk maternal-fetal expert Dr. Julianne Toohey, gastrointestinal endoscopy specialist Dr. John Lee, and pancreatic cancer surgeons Dr. Aram Demirjian and Dr. David Imagawa.

Lee an authority in diseases of the liver, pancreas, bile ducts and gallbladder examined Maglinte using endoscopic ultrasound. His findings led to a biopsy of her pancreas. He also implanted a bile duct stent to alleviate her jaundice. Although complex, each procedure was minimally invasive and safe for the baby.

But the diagnosis was daunting: Maglinte had an aggressive form of pancreatic cancer. Its strange that she would have had cancer at that age, Lee notes. Statistically, almost all pancreatic cancer patients are older than 45, with the average age at the time of diagnosis being 70. Maglinte was only about half that.

She was devastated. I kept walking around the halls of the maternity ward saying: How the heck did I get here? This is crazy.

There werent many options. Early delivery meant the baby would not have survived, as I was only 19 weeks pregnant, Maglinte explains.

But she also had two children at home to consider. She and her husband, Dennis, discussed it. He said it was my choice, Maglinte relates. I wanted to fight for her, but I also needed to fight for myself.

The physicians worked together to save both mother and child. Taking care of a high-risk patient with cancer involves careful communication with the whole team and, of course, the patient, Toohey says. Karalayne was very involved in decision-making.

A week after the diagnosis, Demirjian operated, performing a seven-hour Whipple procedure, or pancreaticoduodenectomy, to remove the tumor. But Maglinte didnt have chemotherapy, which would have jeopardized Emlees survival.

We watched the babys growth and ended up with a planned delivery at 39 weeks, Toohey recalls. This is rather unusual with cancer patients we usually deliver several weeks earlier in order for chemo or other treatment to begin as soon as possible.

Happily, 7-pound, 1-ounce Emlee was born without complications. Her mom says shes a little lifesaver because only 20 percent of pancreatic cancers are diagnosed early, mainly because symptoms abdominal or mid-back pain, jaundice, weight loss and indigestion can overlap with those of other conditions. Without Emlee, I probably wouldnt have reacted to my symptoms the way I did, Maglinte says.

She and Emlee received the kind of advanced care thats usually only available at an academic medical center like UCI.

Oncologist Dr. Rita Mehta, whose research over the past 15 years has led to many advances in treating some of the most aggressive forms of breast cancer with lifesaving results, stands in front of an inspirational quilt that hangs on display at the UCI Breast Health Center in Orange. Photo: Steve Zylius / UCI

Oncologist Mehta, who joined the faculty in 2001 as a clinical professor of medicine, lauds the university, saying, One of the great things is that not only can you do research here, but you can apply what you learn from that research to treat high-risk pregnancies with cutting-edge techniques. Thats not possible in a community hospital setting.

That distinction was as important to Michelle Clark-Salib as it was to Maglinte. In 2012, at age 28, she was diagnosed with an aggressive 8-centimeter breast tumor. After undergoing nearly three months of chemotherapy with a community oncologist in Riverside, Clark-Salib found out that she was 23 weeks pregnant with her son Caleb.

The North Fontana woman consulted with an obstetrician, who discovered that her amniotic sac contained almost no fluid, a side effect of one of her chemo drugs that posed a serious threat to the developing fetus. The doctor sent Clark-Salib to the high-risk maternalfetal physicians at UCI Health, where he had trained as a resident.

Mehta, an international expert in metastatic breast cancer, eventually took over the case. Michelle is an amazing young woman, Mehta says. She was at a very critical stage when she came to us but wanted to save her babys life and her own life. Abortion wasnt an option for her.

The drug that was causing the amniotic fluid problem was discontinued, and Mehta devised a modified cancer treatment plan that avoided the more toxic drug Herceptin until the infant arrived.

As soon as the baby was delivered, we put her back on a chemotherapy regimen, and her cancer went into complete remission, Mehta recalls. Caleb was born healthy at 37 weeks gestation.

Mehta finds this an exciting time in her field: Survival rates are so much improved since I began working in oncology. Sometimes the steps are small; sometimes theyre bigger. But overall, each step adds to the next, and rates keep improving.

Over the last 15 years, her research has led to many advances in treating the most aggressive breast cancers. In a groundbreaking study published in 2012, Mehta showed that a combination of the drugs anastrozole and fulvestrant was superior in controlling cancer and improving patient survival to anastrozole alone or anastrozole followed by fulvestrant in treating hormone receptor-positive metastatic breast cancer in postmenopausal women. A long-term update of the study, published in March 2019 in The New England Journal of Medicine, confirmed the increase in five-year survival rates for advanced breast cancer patients.

Mehta was also one of the first to use chemotherapy combined with Herceptin on women with breast cancer before surgery rather than only after to help shrink tumors.

Now, more than seven years after Calebs birth, Clark-Salib remains cancer-free. And Maglinte has been cancer-free for more than six years.

I dont know what I would have done without UCI, Maglinte says. We were at the right place at the right time with the right teams. Everything just fell into line. Everyone we needed to be there was there.

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Letters to the editor tile(Photo: Argus Leader)

Letters to the Editor for Feb. 2:

The Hippocratic oathis a commitment all physicians make to our patients. Whether its ordering tests, recommending treatment plans, or communicating with families, we take this oath into consideration in all aspects of our medical practice. As a pediatrician, I take very seriously the need to protect children. It is the foundation of our profession. However, I cannot ignore the devastating consequences to children that are being proposed in HB 1057 all in the name of protecting children."

This bill ignores evidenced-based gender affirming care that is supported by organizations such as the American Academy of Pediatrics, American Medical Association, and American Academy of Child & Adolescent Psychiatry and criminalizes doctors who provide this care to patients. Studies show that gender affirming care is linked with lower odds of suicidal thoughts over the lifetime of young transgender people and the physical effects of supportive treatment are not permanent if treatment is stopped. Complex care decisions for the transgender and gender diverse youth are not made on a whim. It is a process that requires lengthy evaluation and counseling with the child, parents, and the medical team to ensure that the wellbeing of the child is at the core of the decision. Turning over that sacred and confidential trust to the legislature to determine is not good medicine.

As a pediatrician and advocate for all children, I believe HB 1057 is harmful to the wellbeing of our children. It creates a precedent where policy makers dictate medical practice that is not based on evidence but on personal opinions. Furthermore, it is government intrusion into the private patient-family-doctor relationship. To support HB 1057 is equivalent to a vote of no confidence in our physicians ability to care for children. It should be rejected by our lawmakers.

Richard Vo, MD, Sioux Falls

I own many guns and am an avid hunter. I am also an honorably discharged Vietnam Veteran (drafted) and very active in South Dakota's National Alliance for Mental Illness (NAMI). With this said I must say I amvery disappointed in our current legislature. I have approached a number of legislators asking if they would sponsor a simple bill. It would prohibit the future sale of any magazine clip which has the capacity to hold more than five bullets. It would not impact the ownership of any firearm or these clips, but would prohibit any future sale. There are similar laws already in place - you must have your shotgun plugged so it can only hold three shells when hunting waterfowl.

We have been fortunate here in South Dakota not to have encountered any mass shootings . If limiting the number of bullets within any firearm saves one life, it is good legislation. It is apparent the NRA wields so much power, influenceand money that any common sense gun control legislation is doomed.

I am attempting to do something but cannot find a single sponsor. This discussion must occur before we have our own tragedy. If there is one legislator willing to discuss, please contact me.

David Braun, Pierre

The state legislature is a good few weeks into itssession. Sadly, it appears that this year will follow the usual muddle of bills as some legislators are off to a mean start with the transgender bill (at least Fred Deutsch has moved the proposal out of the bathrooms this year) and the inane ban on a ban of plastic bags.

Come on people!! This state needs to focus on the big deal stuff: increasing Medicaid, providing assistance for the rapidly growing list of nursing home closures, increasing pay for teachers and state employees, looking at alternate ways to provide income to the state such as legalizing sports gambling and hemp production, revamping the trust laws, and yes, just maybe calling for a small personal income tax and (gasp) corporate income tax. One can hope that during the next few weeks, some of this will be accomplished.

And we (thats you and me, folks) can make a difference: get to the cracker barrel sessions, email your legislators, call your legislators. Put pressure on them to do what is important. Let them know we will no longer abide legislators who idle away our tax dollars by squandering time on these waste makers.

Mary Richards, Spearfish

Im a female veteran who served in the United States Army and strongly support Senator Bernie Sanders for President. As a member of the Army Medical Specialist Corps, I cared for active duty and retired military and their families at Walter Reed and Tripler Army Medical Centers, an honor of which I am extremely proud. Military members valiantly don uniforms, in exchange for their lives, to defend our country from harm. Families unselfishly stand by their military members and country.

More than 6,900 US troops have died in Iraq and Afghanistan; over 52,000 have been wounded. Studies have shown 13-30% of returning troops screen positive for PTSD. It is reported that 17 veterans commit suicide every day. Thousands of military families lost a loved one or have a loved one return from deployment who can no longer function as a father, a mother, a son, a daughter, a significant other, due to war-related trauma. These tragedies are largely suffered by us, working class families, the backbone of our military, who believe in defending our country and in politicians who ask of us the ultimate sacrifice.

Senator Sanders understands the weight of such requests. In 2002, he voted against rushing into war with Iraq without proof of weapons of mass destruction. Too many brave lives have been lost or damaged and the human destruction continues to this day. Yet, politicians, Democrats and Republicans, are raising questions about entering into another war with Iran. Who pays for these decisions? Our working-class families do, in life, limb and mind. Senator Sanders has stated, I will do everything I can to stop a war with Iran."

Senator Sanders will honor our military, during and after active service. I stand with Senator Sanders for President.

Mary Dugan, Hot Springs

I am compelled to write this letter as I just read the disturbing news of another inmate succeeding in committing suicide by hanging under the watch of our county jail system. I now know of twohuman beings in eightmonths committing suicide by hanging in our county jail. My niece succeeded in May of 2019. I have to ask myself:how can this happen?Are there not enough qualified staff to protect those that might be a danger to themselves? Orare some of these mentally ill people thought of like "frequent flyers" that they don't need to be watched?

I am saddened for the family of the man who died in jail on Wednesday Jan 29. I can not tell you how much a family is hurt from the issue of suicide. Please if you know someone who is struggling with mental illness, please say something to anyone. Let it be known. For those who are struggling please get some help anywhere. Ask someone. I pray for peace.

Kaylynn Montis, Sioux Falls

Once again, the South Dakota Legislature is obsessed with all things transgender. After failed attempts to ban transgender kids from bathrooms and sports, Representative Fred Deutsch is attempting to ban those same kids from appropriate medical treatment. He found a gullible House after his exhaustive review of the internet and comparing treatments the what the Nazis experiments did in Auschwitz.

Maybe its time for a reality check:

1) Are there any doctors or surgeons in South Dakota that perform gender confirming surgeries? According to Blue Cross/Blue Shield the answer is NO.

2) Are there ethical guidelines for gender confirming treatments that must be followed for any counselor, doctor, endocrinologist, or surgeon? The answer again in YES. If Representative Deutsch had done his homework, he would have learned of International guidance called WPATH that among other things requires the transgender patient to undergo counseling and be certified by two counselors with a diagnosis of gender dysphoria.

3) Are there counselors certified under the WPATH guidelines? YES, but, there are few certified counselors available. As an example, there are only two in Sioux Falls; however, there are many that advertise services to transgender individuals. In my opinion, this is the area where there are significant weaknesses and the State should be more active is policing those that offer services without certification.

4) Who can administer hormone therapy necessary under the guidelines? Appropriately the answer is an endocrinologist. Again, looking at Sioux Falls, there are a very limited number of these doctors available with months long waiting lists for treatment and who also require referrals from the counselors noted previously.

5) What else should you know? Hormone blockers are provided to identified youth as they enter puberty to pause puberty and is totally reversible. This is a collaborative effort between the patient, their parents, doctors, and counselors. A reasonable person should be able to draw the conclusion that there is an extremely long process before a minor could receive any treatment.

6) Are there other requirements to legally change names and gender? YES, but this also requires some of the services listed above.

These kids have the highest risk of harming themselves or committing suicide. They are at the highest risk for being homeless by outing themselves to their parents. They are the highest risk group subject to bullying as well. You should understand that these individuals dont want to be outed because of the risks of abuse. If you think undergoing treatment is a choice you should know that this is probably the hardest choice a person could ever make. The question you should ask yourself and your representatives is would you rather provide treatment to these individuals or risk a child committing suicide?

Once again Representative Deutsch is leading the charge to discriminate against a small population of vulnerable kids. We should be better than this.

Steve Marty, Sioux Falls

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If you have a kid who wets the bed, you may be wondering if theres an underlying psychological issue. Why do kids wet the bed? Is it because they have some hidden trauma affecting their bladder control? Heres what you should know about bed-wetting and how to support a child who cant seem to wake up dry.

Its very common for kids to pee in their beds at night. According to Mayo Clinic, Bed-wetting also called nighttime incontinence or nocturnal enuresis is involuntary urination while asleep after the age at which staying dry at night can be reasonably expected. Dont be alarmed. In fact, at any given time, about 5 million children (in the US alone) have problems with bed-wetting. Not surprisingly, there are many potential reasons some kids cant stay dry overnight.

Here are some possible factors:

At age five, approximately 15% of kids tend to wet the bed according to the Nationwide Childrens Organization. Boys are two times more likely to have bed-wetting problems than girls. As kids grow older, most have fewer instances of waking up wet. By the time they reach age 14, only 1-2% of kids still struggle with nighttime bladder control.

Waking up wet can be traumatic for any kid, so resist the urge to scold your little one for wetting the bed again. They arent doing it on purpose and need your support and understanding. Reassure them that they havent done anything wrong and help them understand that many children wake up wet in the night. Soothe them instead of punishing them and remind yourself that their body may not yet have the physical maturity required to remain dry through the night.

You can also try the following to support your child through this challenging stage of life. You can take many steps to help them, depending on the underlying issue. If youre looking at their water intake or drinking habits, consider:

If your childs bedwetting is suspected to be tied to their behavior, you can:

If youre exhausted by constantly waking in the night and cleaning up wet bedding, take comfort in knowing most kids grow out of this behavior by the time they reach age seven. If your child doesnt grow out of it by then, or shows other symptoms of an underlying problem, it may be time to talk to your doctor.

The American Family Physician Organization says administering bed-wetting medication from your pediatrician should be your last resort, Medicines arent a cure for bed-wetting. The medicines work in two ways. One kind of medicine helps the bladder hold more urine, and the other kind helps the kidneys make less urine. The medicines may have side effects.

Take your child to the pediatrician if you notice that her nighttime urinating is accompanied by other behaviors or problems, such as pain while peeing, sudden bed-wetting after months of staying dry, swelling in the ankles and feet or snoring loudly at night. There may be a treatable medical condition at the root of the cause.

Wetting the bed is something many kids and parents go through. The key is to face the challenge together, with plenty of support, encouragement, and patience until the phase passes.

Want to know more about when kids stop wetting the bed?

Bed-wetting in kids: Why it happens and what to doBedwetting: 5 Common Reasons Why Children Wet the BedWhy Does My Child Wet the Bed?Bed-wetting

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Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

Crystal Nguyen, now 20, was first diagnosed with acute myeloid leukemia at age 12. She found a stem-cell match for a needed bone-marrow transplant through the international cord blood bank.Handout

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

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WARSAW (AFP) Submerged in liquid nitrogen vapour at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukaemia and lymphoma, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industrys leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States (US), a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

Mum-of-two Teresa Przeborowska has firsthand experience.

At five-years-old, her son Michal was diagnosed with lymphoblastic leukaemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sisters stem cells into Michals bloodstream. It was not quite enough for Michals needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, is now flourishing, both intellectually and physically, his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, each container holds up to 10,000 blood bags. Safe and secure, they wait to be used in the future, its Head Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the blood will be ready to use without the whole process of looking for a compatible donor and running blood tests, the biologist told AFP.

For families who have paid an initial nearly EUR600 (USD675) and then an annual EUR120 euros to have the blood taken from their newborns umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

Bone marrow pioneer in Poland Haematologist Wieslaw Jedrzejczak describes promoters of the treatment as sellers of hope, who make promises that are either impossible to realise in the near future or downright impossible to realise at all for biological reasons.

He compares them to makers of beauty products who swear their cream will rejuvenate the client by 20 years.

Various researches is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

There is a list of almost 80 diseases for which stem cells could prove beneficial, US Haematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood programme Vitalant in New Jersey, told AFP.

But given the present state of medicine, they are effective only for around a dozen of them, like leukaemia or cerebral palsy, he said.

Its not true, as its written sometimes, that we can already use them to fight Parkinsons disease or Alzheimers disease or diabetes.

EuroStemCell also cautions against private blood banks that advertise services to parents suggesting they should pay to freeze their childs cord blood in case its needed later in life.

Studies show it is highly unlikely that the cord blood will ever be used for their child, the network said.

It also pointed out that there could be a risk of the childs cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most European Union (EU) countries however permit it while imposing strict controls.

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of UER2,500 euros upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with PLN2million (around EUR450,000, USD525,000), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35 per cent of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed EUR63 million to the firm, PBKMs Chief Executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.

Read more:
Europe's guardian of stem cells and hopes, real and unrealistic - Borneo Bulletin Online

Recommendation and review posted by Bethany Smith

Thought leaders in cancer cell and gene therapy research will share the latest advances, address the greatest challenges and showcase the most innovative programs in progress today.

Alliance for Cancer Gene Therapy (ACGT) will host its inaugural ACGT 2020 Cancer Summit on April 16, 2020 at the Alexandria Center for Life Science in New York City. The ACGT 2020 Cancer Summit which launches ACGTs 20th anniversary will bring together researchers, companies, investors and advocates in cancer cell and gene therapy to discuss the latest advances, with a focus on combating solid tumors.

A partial list of ACGT 2020 Cancer Summit speakers includes:

Presenting sponsors include Alexandria Real Estate Equities, Inc./Alexandria Venture Investment. Additionally, STAT will be a media partner for the ACGT 2020 Cancer Summit. For more sponsorship information and early bird registration, please visit https://summit2020.acgtfoundation.org

Media Registration

Media registration is free of charge for all valid press card holders or via provision of formal journalist credentials. Register early by contacting ACGT.pr@HDMZ.com to begin receiving advance meeting materials, media alerts, and access to meeting presenters.

About Alliance for Cancer Gene Therapy

For nearly 20 years, Alliance for Cancer Gene Therapy (ACGT) has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Founded by Barbara and Edward Netter after their daughter-in-laws death from breast cancer, ACGT funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. One hundred percent of all public funds raised by ACGT directly support program and research, thanks to separate funding to support administrative expenses.

For more information, visit acgtfoundation.org, call 203-358-5055, or join the ACGT community on Facebook, Twitter, Instagram and YouTube.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200203005084/en/

Contacts

Media Inquiries: Emily Maxwell 312-506-5220emily.maxwell@hdmz.com

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Save the Date: Alliance for Cancer Gene Therapy 2020 Cancer Summit to be Held in New York City - Yahoo Finance

Recommendation and review posted by Bethany Smith

The cell and gene therapy sector is booming. At the end of 2019, there were 1,069 registered clinical trials in the field. By 2024, the cell and gene therapy market is estimated to reach revenues of $6.6B (5.9B). But there is a problem. As a result of the rapid growth of gene and cell therapies, the demand for plasmids is skyrocketing. And the industry is struggling to meet it.

Plasmids are the key building blocks needed to manufacture viral vectors, which are the most common strategy to develop gene therapies, explains Stefano Baila, Director of Operations and Business Development at Anemocyte. Plasmids are not only crucial for the development of gene therapies, but also for therapies that involve the genetic modification of cells, such as CAR-T. This has greatly increased the demand for plasmids and I would say that the industry was probably not ready to address this demand.

While the industry is struggling with the lack of plasmids on the market, another challenge is quality. Those providers that can meet the high-quality requirements for plasmids have long waiting lists of up to 12 months, says Baila. For companies moving into phase 3 or commercial production, it becomes crucial that the good manufacturing practice (GMP) requirements are met.

But another result of the rapidly evolving gene and cell therapy space is the fact that the regulatory requirements are not quite up to scratch. The regulatory framework around plasmid production is very confusing for the industry at the moment, says Baila. The main guidelines refer to the quality of the product, but the level of quality remains open to interpretation. More clarity would definitely help once and for all to define the exact quality levels required at different stages of drug development.

Consequently, plasmid providers have to be able to address all quality levels required at each stage of drug development. As the first biotech manufacturing organization (BMO) worldwide, Italian company Anemocyte has met this challenge by focusing greatly on the industrys needs. Their keyword is flexibility.

Before starting their work on plasmid manufacturing, the team spent several months interviewing companies about their difficulties and needs regarding the bottleneck in plasmid production and regulatory issues.

For us, it was key to understand the needs of the industry and find a possible solution, Baila explains. Our research resulted in a brand new facility, which is designed with adaptable manufacturing spaces that enable flexible time management. This ensures that the manufacturing process continues to roll without creating a bottleneck.

As a next step, the Anemocyte team had to decide whether to use a classified cleanroom or just a regular lab for the manufacturing process. We decided to keep the bar pretty high, so we are working with a cleanroom facility where we apply the GMP standard, says Baila. We maintain a high quality and also address the time issues that all companies seem to share as their main challenge in cell and gene therapy development.

The flexibility of its manufacturing facility allows the Anemocyte team to easily adapt to its customers needs. Our customers have control over what we do, explains Marco Ferrari, CEO of Anemocyte. They have the opportunity to be involved in the process, and decisions and actions are discussed and shared with them to ensure their product is produced at the high standard they expect.

Moreover, Anemocyte pays attention to new technologies and innovations. The fast evolution of the cell and gene therapy industry greatly increases the demand for new solutions, Ferrari explains. Staying on top of innovation is therefore mandatory today. Our approach is to stay ahead of the trends and be capable of deploying useful solutions for our customers.

Anemocytes manufacturing facility is built in such a modular way that it can be replicated and adapted to meet the rising demand for plasmids. This, as Ferrari puts it, ensures that the Anemocyte team will not be caught off guard when more companies come knocking at our door. This is an advantage for long-term customers because the manufacturing facility can be duplicated and built according to the customers needs.

As the worlds first BMO, Anemocyte pays specific attention to innovation and the ongoing trends in the industry. One of the emerging trends is the use of nonviral vectors for the development of gene therapies.

Even in the nonviral approach, plasmids play a key role, explains Baila. A part of the nonviral strategy is the transfer of plasmids into cells via mechanical or chemical methods. So, in one way or another, plasmids will always be needed.

Our investment in the nonviral gene modification space is an example of how we are tackling potential future trends that are still under the radar or explored at an academic level, adds Ferrari.

Dont sit on a waiting list to get your plasmids produced! Get in touch with the team at Anemocyte or learn more about the company and the development of plasmids for cell and gene therapies here!

Images via Shutterstock.com

Author: Larissa Warneck, Science Journalist at Labiotech.eu

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How Can We Tackle the Bottleneck in Plasmid Production for Gene... - Labiotech.eu

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$250,000 in Vilcek Foundation Prizes awarded to Xiaowei Zhuang, Kivan Birsoy, Viviana Gradinaru, and Martin Jonikas.

NEW YORK, Feb. 3, 2020 /PRNewswire/ -- The Vilcek Foundation is pleased to announce the winners of the 2020 Vilcek Prizes in Biomedical Science. Awarded annually, the Vilcek Prizes in Biomedical Science honor the contributions of immigrants to scientific research, discovery, and innovation in the United States. The Vilcek Prizes in Biomedical Science are a part of the Vilcek Foundation's prizes program, which honors the contributions of foreign-born persons in the arts, sciences, and humanities in the United States.

The Vilcek Prize in Biomedical Science recognizes an immigrant scientist for outstanding career contributions to biomedical science and global scientific research. The prize comprises an unrestricted cash prize of $100,000 and a commemorative award. The winner of the 2020 Vilcek Prize in Biomedical Science is Xiaowei Zhuang, the David B. Arnold Jr. Professor of Science at Harvard University and a Howard Hughes Medical Institute Investigator.

"Honoring the achievements of immigrant scientists and to articulate their enormous role in upholding the United States' world leadership in science is especially important at this juncture in view of the current administration's efforts to curtail immigration to this country," said Jan Vilcek, cofounder and chairman of the Vilcek Foundation. "Xiaowei Zhuang exemplifies a foreign-born scientist whose work has demonstrably shaped the further advancement of scientific technologies and discovery in the United States. The imaging tools she has developed have changed how we see and understand the structures and basic functions of living organisms at a molecular level, fundamentally expanding research horizons and possibilities, and cementing the United States as a global leader in science."

Xiaowei Zhuang developed methods to directly observe individual molecules in cells at high resolution and at genomic scale. She invented STORM, a super-resolution imaging method that surpasses the diffraction limit of conventional light microscopy and enables light microscopy at molecular-scale resolution. Using STORM, Zhuang has unveiled novel structures in cells, such as a periodic membrane skeleton in neurons, which provides a platform for signal transduction and lends mechanical stability. Zhuang invented a single-cell transcriptome and genome imaging method, MERFISH, which allows thousands of genes to be simultaneously imaged and quantified in individual cells. MERFISH is a key tool in the Human Cell Atlas projectan international effort aimed at cataloguing cell types that constitute humans. Zhuang has used this tool to identify novel types of neurons in the mouse brain and uncovered insights into the neuronal control of behavior. Zhuang was born in Rugao, China.

The Vilcek Prizes for Creative Promise in Biomedical Science are awarded to young immigrant scientists whose early career work represents a profound advance in their respective fields. Each prizewinner receives a $50,000 cash prize and a commemorative award. Kivan Birsoy, Viviana Gradinaru, and Martin Jonikas are the winners of the 2020 Vilcek Prizes for Creative Promise in Biomedical Science.

Kivan Birsoy has fashioned tools to probe the complex metabolism of cancer cells. Using CRISPR, Birsoy demonstrated how mitochondriathe powerhouses of animal cellssupport tumor growth. He found that the synthesis of the amino acid aspartate rather than energy generation is key to mitochondria's ability to fuel tumors. He custom-designed instruments called Nutrostats for the long-term laboratory culture of cancer cells under nutrient levels closely mimicking natural conditions. Combining the Nutrostats with DNA barcoding and CRISPR-based genetic screens, Birsoy showed that a rare but deadly type of lymphoma requires LDL-cholesterol for survival. His work has identified altered tumor metabolism as a potential therapeutic target in cancer. Birsoy was born in Izmir, Turkey.

Viviana Gradinaru has developed advanced tools in optogeneticsan approach that uses light to control brain cellsto unearth deep insights into the brain. Her work has uncovered circuits that may underpin sleep disturbances in neuropsychiatric disorders. She has also engineered vehicles for gene therapy that, when injected into the bloodstream of rodents, cross the blood-brain barrier and deliver target genes to the brain. Gradinaru also developed a tissue-clearing technique called PARS-CLARITY to render the entire body of a rodent transparent for fine-scale visualization and mapping of circuits as well as gene expression analysis. Her work could boost the development of gene therapies for an array of human disorders. Gradinaru was born in Vaslui, Romania.

Martin Jonikas has used molecular biology to advance the single-celled green alga Chlamydomonas as a widely used model for photosynthesisa biochemical process at the heart of global food production. Jonikas and colleagues have unraveled the molecular makeup, structure, and assembly of pyrenoids, which are cellular compartments found in photosynthetic organisms that can absorb planet-warming carbon dioxide. This work could aid efforts to engineer pyrenoids into species that lack them, paving the way toward environment-friendly food production and helping to combat climate change. Jonikas was born in Paris, France.

The foundation has also announced the recipients of the 2020 Vilcek Foundation Prizes in Literature, and the recipient of the 2020 Vilcek Prize for Excellence in the Administration of Justice. All of the prizewinners will be celebrated at the Vilcek Foundation Prizes Gala in New York in April 2020.

The Vilcek Foundation

The Vilcek Foundation was established in 2000 by Jan and Marica Vilcek, immigrants from the former Czechoslovakia. The mission of the foundation, to honor immigrant contributions to the United States, and more broadly, to foster appreciation of the arts and sciences, was inspired by the couple's respective careers in biomedical science and art history. The foundation awards annual prizes to immigrant biomedical scientists and artists, sponsors cultural programs, and manages the Vilcek Foundation Art Collections. To learn more, please visit vilcek.org.

Contact:Elizabeth BoylanThe Vilcek Foundation212-472-2500 or elizabeth.boylan@vilcek.org

View original content to download multimedia:http://www.prnewswire.com/news-releases/vilcek-prizes-recognize-immigrant-scientists-for-the-development-of-tools-and-technologies-advancing-scientific-discovery-300997189.html

SOURCE The Vilcek Foundation

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Vilcek Prizes Recognize Immigrant Scientists for the Development of Tools and Technologies Advancing Scientific Discovery - BioSpace

Recommendation and review posted by Bethany Smith

Somerset Feb 3, 2020 (Thomson StreetEvents) -- Edited Transcript of Catalent Inc earnings conference call or presentation Monday, February 3, 2020 at 1:15:00pm GMT

* John R. Chiminski

Catalent, Inc. - Chairman & CEO

* Wetteny N. Joseph

Catalent, Inc. - Senior VP & CFO

UBS Investment Bank, Research Division - Senior Equity Research Analyst of Healthcare Life Sciences

Robert W. Baird & Co. Incorporated, Research Division - Senior Research Associate

* Jacob K. Johnson

William Blair & Company L.L.C., Research Division - Partner & Healthcare Services Analyst

* Tycho W. Peterson

Ladies and gentlemen, thank you for standing by, and welcome to the Catalent Second Quarter Fiscal Year 2020 Earnings Conference Call. (Operator Instructions) Please be advised that today's conference is being recorded. (Operator Instructions)

I would now like to hand the conference over to your speaker today, Paul Surdez, Vice President, Investor Relations. Thank you. Please go ahead, sir.

Good morning, everyone, and thank you for joining us today to review Catalent's Second Quarter Fiscal Year 2020 Financial Results. With me today are John Chiminski, Chair and Chief Executive Officer; and Wetteny Joseph, Senior Vice President and Chief Financial Officer.

In addition to reviewing our second quarter earnings release issued earlier this morning, we will also refer you to our other press release issued today regarding our agreement to acquire cell therapy leader, MaSTherCell. Please see our agenda for this call on Slide 2 of our supplemental presentation, which is available on our Investor Relations website at http://www.catalent.com.

During our call today, management will make forward-looking statements and refer to non-GAAP financial measures. It is possible that actual results could differ from management's expectations. We refer you to Slide 3 for more detail. Slides 3, 4 and 5 discuss the non-GAAP measures, and our just-issued earnings release provides reconciliations to the nearest GAAP measures. Catalent's Form 10-Q, to be filed with the SEC later today, has additional information on the risks and uncertainties that may bear on our operating results, performance and financial condition.

Now I would like to turn the call over to John Chiminski.

John R. Chiminski, Catalent, Inc. - Chairman & CEO [3]

Thanks, Paul, and welcome, everyone, to the call. In addition to reporting strong Q2 results, we're excited to announce this morning our plan to further expand our biologics footprint by acquiring MaSTherCell, a leader in cell therapy development and manufacturing.

Before reviewing the strategy behind adding MaSTherCell to the Catalent family, let me summarize our financial highlights from the second quarter. As you can see on Slide 6, our revenue for the second quarter increased 16% as reported or 17% in constant currency to $721 million, with 7% of the constant currency growth being organic, which is above our expectations for the long-term organic growth of our base business.

Our adjusted EBITDA of $171 million for the quarter was above the second quarter of fiscal year 2019 on a constant currency basis by 16%, with 5% being organic. Our adjusted net income for the second quarter was $72 million or $0.45 per diluted share, unchanged from the per share adjusted net income in the prior fiscal year.

Three of our 4 reporting segments had strong performances as Biologics, Softgel and Oral Technologies and Clinical Supply Services each contributed to the organic revenue and adjusted EBITDA growth, partly offset by headwinds in our Oral and Specialty Delivery segment. Wetteny will detail these results later in the call.

Now moving on to the operational update. First, we announced 2 important executive appointments in January that provide additional depth and breadth to our leadership team. We recruited Karen Flynn to return to Catalent after 10 years of leading operations and commercial activity for a well-respected biopharma services company to be President of our Biologics segment and our Chief Commercial Officer. Karen, who is replacing the retiring Barry Littlejohns, will execute our biologics strategy and further expand our Biologics drug substance, drug product and gene therapy businesses. We also recruited another former Catalent executive with decades of experience in the biopharmaceutical industry, Ricci Whitlow, as our President of Clinical Supply Services in place of the retiring Paul Hegwood. In addition to growing our CSS business with our traditional customer base, she will be focused on growing its footprint through cross-selling opportunities with our Biologics and other long-cycle businesses. Karen and Ricci, like Barry and Paul before them, report to our COO, Alessandro Maselli. They replace distinguished leaders who are celebrated here at Catalent for growing their businesses and for their tireless efforts to help establish our patient-first culture.

Next, last week, the Catalent Board of Directors approved the deployment of additional capital for further expansion of our gene therapy commercial facilities at BWI, which expansion will support operations on the BWI campus as well as our other gene therapy facilities in BioPark, Rockville and Gaithersburg. This investment is above and beyond the CapEx previously approved for the build-out of the 10 suites in our BWI facility, all of which are on track to be operational at the end of this calendar year. The additional CapEx approved last week will allow us to achieve higher revenue potential from the Paragon acquisition than anticipated at the time of the original acquisition last May once all the projects are completed.

Additionally, early last month, we took ownership of Bristol-Myers Squibb's oral solid biologics and sterile product manufacturing and packaging facility in Anagni, Italy, which we had agreed to acquire in June. This multipurpose site enhances our global network and provides us drug product sterile fill/finish capacity and oral solid-dose manufacturing in Europe and comes with an agreement to continue to manufacture BMS's current product portfolio at the site. The Anagni facility expands our European capabilities in biologics drug product, solid oral dose manufacturing and packaging to accelerate development programs and provides greater commercial supply capacity.

The acquisition of Anagni is another example of our progress in realizing our global biologics strategy, which continues to develop and strengthen across our network. As an additional example, I'm pleased to announce that the Bloomington site received yet another commercial product approval in January, bringing its total to 22 versus the 12 it was producing at the time of the acquisition, with several additional launches on the horizon. The previously announced $200 million investment in Bloomington and Madison are progressing according to plan and will help us serve the existing pipeline of late-stage clinical work and other opportunities for these high-margin sites.

Another important element of our biologics strategy is our entrance into the gene therapy space last year. The acquisitions of Paragon Bioservices and related gene therapy assets provided Catalent with new expertise and capabilities in one of the fastest-growing techniques for therapeutic intervention today and position us for accelerated long-term growth. The integration of these gene therapy assets into the Catalent portfolio is progressing ahead of our expectation and has been a key contributor to our strong year-to-date financial results. The CapEx approval I previously highlighted was supported by this early outperformance as well as by research remission from an independent third-party consultant, which indicates the gene therapy pipeline will continue to increase much more rapidly than the manufacturing assets needed to service the demand.

Paragon provided us with a platform for development of an expanded offering in biologics, enabling entry into technology categories adjacent to the development and production of viral vectors for gene therapies. The success we've experienced thus far with Paragon provides us with the confidence and blueprint to further expand our biologics offering into cell therapy, which we are announcing this morning.

Please turn to Slide 7 for an overview of our agreement to acquire MaSTherCell, a technology-focused cell therapy development and manufacturing partner to cell therapy innovators. MaSTherCell's service offerings include the development and manufacture of both autologous and allogeneic cell therapies as well as a variety of related analytical services. It has worked with a range of therapies, including those based on the so-called CAR-T cells, tumor-infiltrating lymphocytes as well as T cell receptors and other cell types.

MaSTherCell, which was founded in 2011, has sites in Belgium and Texas. Its current operating facility near Brussels provides preclinical and clinical stage services, and MaSTherCell is in the process of building a commercial-scale production and fill/finish facility nearby, which is expected to open in late 2021. MaSTherCell is also in the final stages of completing the build-out of a preclinical and clinical stage facility near Houston, Texas and has future plans to expand into commercial there as well.

Cell therapy, like gene therapy, is attracting enormous funding as both the number of active programs and the level of funding have rapidly expanded over the last 5 years. There are now more than 500 public and private companies with cell therapy programs and hundreds of active cell therapy-based investigational new drug applications. Much of the focus today is in oncology, but we're seeing applications expand in other therapeutic areas, such as autoimmune diseases, cardiology and neurology. Our research indicates that the cell therapy pipeline is growing in the mid-teens range with over 800 cell therapy assets in the pipeline today and also estimate cell therapy manufacturing to be approximately 65% outsourced, which is comparable to viral vectors.

Also, similar to viral vector manufacturing, cell therapy capacity is scarce, and the trend of demand outstripping supply is projected to become more acute despite investments in additional capacity being made across the industry. We see MaSTherCell as a complementary addition to our gene therapy capabilities and the rest of our Biologics portfolio.

We also believe that MaSTherCell will be a strong, strategic fit for Catalent as we're well positioned to combine MaSTherCell's team of experts and differentiated capabilities with our extensive resources and our significant experience in scaling new platforms to help MaSTherCell build out its development and commercial manufacturing capabilities. Furthermore, we believe MaSTherCell rounds out our program to be the leader in gene and cell therapy, creating deeper and broader relationships with customers, and like we've seen with Paragon, open up cross-selling opportunities across Catalent's other technology platforms.

From a structural perspective, this is an all-cash transaction with a total purchasing price of $315 million subject to customary purchase adjustments. Catalent expects to finance this transaction with either a partial drawdown of its revolving credit facility with proceeds from a possible future incremental capital raise. Any such raise may also include funds for capital expenditures in support of our gene therapy programs and other strategic initiatives.

Slide 8 illustrates how our actions continue to fundamentally transform our business and increase our share of the R&D pipeline by significantly increasing our exposure to the faster-growing area of the industry that is biologics. We've done this through significant organic and inorganic investments, putting to work nearly $3 billion over the last 5 years.

In the 12-month period ended December 31, our Biologics segment represented 27% of our portfolio. In the quarter we're reporting today, it's now just over 30%. And when factoring in our long-term organic revenue growth guidance of 6% to 8%, combined with strategic acquisitions like MaSTherCell and Anagni, we believe we're on pace for 50% of our revenues to be driven from Biologics segment by the end of fiscal 2024 with total company revenues projected to be approximately $4.5 billion. Given the greater margin contributions from our Biologics segment, we believe adjusted EBITDA margins in 2024 will expand to at least 28%, up approximately 300 basis points from our expected levels in 2020.

We're proud that the combination of organic and inorganic investments we're making in biologics is already delivering substantial benefits to patients. We believe our strategy that drove us to uniquely combine capabilities to support the fastest-growing areas of drug development with Catalent's historical leadership and deep expertise in global contract drug manufacturing will continue to create significant value for our company, our customers and our shareholders.

Now I'll turn over the call over to Wetteny, who will take you through our second quarter financial results and the details related to our updated financial guidance.

--------------------------------------------------------------------------------

Wetteny N. Joseph, Catalent, Inc. - Senior VP & CFO [4]

--------------------------------------------------------------------------------

Thanks, John. I will begin this morning with a discussion on segment performance, where both the fiscal 2019 and fiscal 2020 second quarter results are presented on the basis of the revised reporting segments we introduced last quarter. Please turn to Slide 9, which presents our Softgel and Oral Technologies business. As in past earnings calls, my commentary around segment growth will be in constant currency.

Softgel and Oral Technologies revenue of $267.9 million increased 3% during the quarter, with segment EBITDA increasing 19%. After excluding the impact of the October 2019 divestiture of the segment's manufacturing site in Braeside, Australia, segment revenue and EBITDA grew 9% and 24%, respectively. The growth primarily relates to volume increases across the consumer health portfolio within Europe as well as increased demand in the prescription product business in North America, which is partially attributable to recently launched products.

Revenue in the consumer health business also increased in North America and Latin America due to the prior year shortage in ibuprofen API supply. Additionally, the strong segment EBITDA performance was driven by improved capacity utilization and favorable product mix across the network.

Slide 10 shows that our Biologics segment recorded revenue of $225.2 million in the quarter, which is up 66% versus the comparable prior year period, with segment EBITDA growing 61% quarter-over-quarter. Note that a large portion of both the revenue and the segment EBITDA growth was inorganic and driven by the gene therapy acquisitions, which contributed 56 percentage points to revenue and 49 percentage points to EBITDA growth. Excluding acquisitions, the segment recorded organic revenue growth of 10% in the second quarter and segment EBITDA growth of 12%.

Recent investments in our Biologics business continued to translate into growth during the second quarter as we recorded strong growth in drug product volumes in the U.S. As a reminder, drug substance revenue continues to be impacted by the completion of a limited-duration customer contract, which had a particularly high drop-through of EBITDA, following the completion of the client build-out of its own capacity. The customer's strategy to move its production in-house was fully contemplated when we entered into the contract, and the precise timing was less defined given typical production complexities. We continue to expect this to be a comparison headwind for our drug substance business for another quarter as we work to onboard new customers to increase our utilization levels. Drug acceptance, after excluding the completion of this noncell line clinical manufacturing contract, also grew year-on-year.

As I mentioned, we just closed on the Anagni acquisition on the 1st of January. As we did not know the timing of the close when we gave initial guidance in August, the site was not included in our original estimate, but is now reflected in our current guidance updated today. As the site is multipurpose, its future financial reporting is likely to be split between Biologics and OSD segments, and we will provide you more details when we report our third quarter.

To close out the commentary on Biologics, I'd like to echo John's excitement about bringing MaSTherCell's cell therapy expertise to Catalent, which enables us to establish a position in this exciting new therapeutic platform and stay at the forefront of bringing new, advanced therapies to scale. Catalent provides MaSTherCell access to growth capital, leverages its functional and system expertise and provides access to additional customers. However, given the company's early stage, MaSTherCell is not expected to provide meaningful EBITDA in the next 2 years as profit generated in its current clinical services will be consumed by its commercial build-out. We expect to provide additional color next quarter following the expected closing of the transaction.

Slide 11 shows that our Oral and Specialty Delivery segment recorded revenue of $143.2 million in the quarter, which is down 7% versus the comparable prior year period, with segment EBITDA declining 28% quarter-over-quarter. While we experienced growth in our orally delivered commercial products, this was more than offset by decreased volumes in the segment's respiratory and ophthalmic specialty delivery platform. This business experienced very strong demand a year ago as it generated revenues in anticipation of new product introductions. However, these NPIs have not yet materialized, creating a headwind for the segment this quarter, which despite expected sequential improvement will result in a year-on-year headwind for the remainder of the year and is factored in our new guidance. Despite the softness we are experiencing this quarter, we believe the OSD segment continues to have a very strong development pipeline, including several late-stage quasi-development programs that will drive future long-term growth.

In order to provide additional insight into our long-cycle businesses, which include Softgel and Oral Technologies, Biologics and Oral and Specialty Delivery, we are disclosing our long-cycle development revenue and the number of new product introductions as well as revenue from these NPIs. As a reminder, these metrics are only directional indicators of our business since we do not control the sales or marketing of these products nor can we predict the ultimate commercial success of them.

For the first 6 months of fiscal year 2020, we recorded development revenue across both small and large molecule of $422.5 million, which is more than 36% above the development revenue recorded in the first half of the prior fiscal year. Additional disclosure on our development revenue is included on our Form 10-Q to be filed today with the SEC. In addition, we introduced 87 new products in the first 6 months of fiscal year 2020, which are expected to contribute approximately $27 million revenue in the fiscal year.

Now as shown on Slide 12, our Clinical Supply Services segment posted revenue of $87.9 million or 9% growth over the second quarter of the prior year, segment EBITDA of $24 million or 15% growth. The strong growth in both revenue and segment EBITDA was driven by strong demand in the segment's storage and distribution and manufacturing and packaging businesses. All the segment revenue and EBITDA growth recorded within CSS was organic.

As of December 31, 2019, our backlog for the CSS segment was $390 million, a 4.5% sequential increase. The segment recorded net new business wins of $104 million during the second quarter, which is a decrease of 2.3% compared to the very high level of net new business wins recorded in the second quarter of the prior year. The segment's trailing 12-month book-to-bill ratio remains at 1.2x.

Slide 13 and 14 contain reference information for our second quarter and year-to-date segment results, both as reported and in constant currency.

Slide 15 provides a reconciliation of EBITDA from operations from the most approximate GAAP measure, which is net earnings. This bridge would assist in tying out our reported figures to our computation of adjusted EBITDA, which is detailed on the next slide.

Moving to adjusted EBITDA on Slide 16. Second quarter adjusted EBITDA increased 17% to $171 million or 23.7% of revenue compared to 23.4% of revenue reported in the second quarter of prior year. On a constant currency basis, our second quarter adjusted EBITDA increased 18%, including 5% organic growth.

On Slide 17, you can see that second quarter adjusted net income was $72 million or $0.45 per diluted share compared to adjusted net income of $65.4 million, also representing $0.45 per diluted share in the second quarter a year ago.

Slide 18 shows our debt-related ratios and our capital allocation priorities. Our total net leverage ratio as of December 31 was 4.2x, which has modestly reduced from the ratio as of the end of the prior quarter. Pro forma for completed acquisitions, our total net leverage ratio was 4.0x, which is an improvement of approximately 1/2 of a turn compared to the ratio at the time we announced the Paragon transaction. Given the free cash flow generation of the company and its growing adjusted EBITDA, the company naturally delevers between 0.5 and 0.75 of a turn per year. Additionally, continued investments in Biologics, including the new CapEx approved by our Board last week for our gene therapy business, led us to increase our fiscal year 2020 projections for CapEx spending. Taking into account customer funding, capital expenditures are now expected to be approximately 13% to 14% of net revenue compared to our initial assumption of 11% to 12% of net revenue. Our capital allocation priorities remain unchanged and focus first and foremost on organic growth followed by strategic M&A.

Now we'll turn to our financial outlook for fiscal year 2020 on Slide '19. As John reviewed in his opening comments, we are raising our financial guidance to reflect the acquisition of Anagni and for the continued growth of the gene therapy business and are also slightly tightening these ranges to reflect the passage of time. No contribution from MaSTherCell is assumed in this revised guidance, which regardless of when it closes will be immaterial to our full year 2020 results.

We now expect full year revenue in the range of $2.87 billion to $2.95 billion compared to our previous guidance of $2.78 billion to $2.88 billion. Note, this new guidance continues to assume organic revenue growth of 4% to 7%. Full year adjusted EBITDA -- for full year adjusted EBITDA, we now expect a range of $711 million to $735 million compared to our previous expectation of $700 million to $730 million. This new range continues to assume our original organic adjusted EBITDA growth assumption of 9% to 12%. Note the greater increase in our revenue guidance relative to our adjusted EBITDA guidance will result in a somewhat lower adjusted EBITDA margin level for 2020 than our original guidance. We now expect adjusted EBITDA margin to increase over fiscal year 2019 results of 23.8% by approximately 100 basis points at the midpoint of the new range versus the previous expectation of an approximate 150 basis point increase. This is largely due to the addition of Anagni, which, as expected, currently has lower utilization levels until it adds more customers.

We're also updating our full year adjusted income guidance to a range of $307 million to $331 million compared to the previous guidance of $300 million to $330 million. We now expect that our fully diluted share count on a weighted average basis for the fiscal year ending June 30 will be in the range of 160 million to 161 million shares, which continues to account the preferred shares we issued in May to fund part of the Paragon acquisition as if they all were converted to common shares in accordance with their terms. We continue to expect our consolidated effective tax rate to be between 24% and 26% for the fiscal year.

Finally, Tom Castellano is also in the room with us today, and I'd like to personally thank him for the outstanding job he has done leading the Investor Relations function for Catalent since our IPO. Tom will continue to add great value to the company in his new leadership role as Global Vice President of Operational Finance and as the finance leader for our Biologics segment. Tom has transitioned his IR responsibilities to Paul Surdez, who joined us last month and many of you know from his time leading Investor Relations at other public health care companies.

Operator, we'd now like to open the call for questions.

================================================================================

Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Tycho Peterson with JPMorgan.

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Tycho W. Peterson, JP Morgan Chase & Co, Research Division - Senior Analyst [2]

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I'll start with MaSTherCell. I know it's a smaller deal than Paragon, but I'm just wondering if you could compare and contrast the 2. How you think about kind of the cellular market versus the gene therapy market? How should we think about CapEx needs? Any customer concentration risks? And then as we think about kind of the longer-term guidance of 10% to 15% for Biologics, what do you think the cellular therapy market opportunity could do to that growth rate?

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John R. Chiminski, Catalent, Inc. - Chairman & CEO [3]

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Sure. A lot there, Tycho. So let me just step back and look at the big picture here. First of all, I think on our acquisition of Paragon in the gene therapy space really gave us the confidence to enter into another very fast-expanding space in cell therapy. When we take a look at the number of cell therapy trials that are ongoing, it actually significantly exceeds those in the gene therapy area, and it's growing kind of in the mid-teens growth rate.

I would say that from an acquisition standpoint, I would say that we have acquired MaSTherCell a little bit earlier in the cycle than we have from a Paragon standpoint. So obviously, a smaller acquisition compared to Paragon, but I would say we're probably catching it 2 to 3 years earlier in the cycle. So they're still early on. They've got a very strong position. I would say they're really the leading stand-alone cell therapy CDMO player, and they've got some tremendous capability. I would say from a customer standpoint, I think it's very similar to our acquisition of Paragon where you've got a couple of marquee base customers there, but then have behind that, a broad slate of overall customers, both in the autologous as well as the allogeneic area.

From a CapEx standpoint, I would say that on a comparative basis to Paragon, they're smaller numbers based upon the overall technology, but it is going to require some additional CapEx for us to build out the commercial facilities that they already have started in the Belgian area as well as the preclinical and clinical facility they have in Houston and an anticipated additional commercial facility there. So we've anticipated that in terms of looking at our CapEx going forward, which Wetteny can further detail out.

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Wetteny N. Joseph, Catalent, Inc. - Senior VP & CFO [4]

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Yes. Tycho, the one thing I would add is, as John mentioned in his prepared commentary, MaSTherCell is in the midst of expanding clinical operations with a new facility in the U.S. in addition to a commercial facility that they're in the middle of in Europe. So as those come on and ramp up, we would expect to attract even more customers into the business as we continue to scale it from a customer standpoint.

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Tycho W. Peterson, JP Morgan Chase & Co, Research Division - Senior Analyst [5]

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Okay. And then just one follow-up on oral and specialty. You talked about the delays in product approvals and maybe some pressure there for the next couple of quarters. I guess should we be modeling that business down then the next couple of quarters? And is -- when does Zydis Ultra start to kind of contribute as well? Is that going to be beneficial at all?

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Wetteny N. Joseph, Catalent, Inc. - Senior VP & CFO [6]

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Yes. So in terms of the remainder of the year, I would say, given my prepared commentary here, I would expect some continued headwind for the OSD segment for the balance of the fiscal year. That's all factored into the guidance that we just gave as well for the year, just giving you some additional color there. Although I would expect the business to show sequential improvement quarter-on-quarter. From a growth rate standpoint, it would still be a headwind for the balance of the year.

In terms of Zydis Ultra , as we've talked about, this is an exciting area for us to expand the base of our Zydis offering to be able to bring on molecules with bigger drug loading than we did before. We have gone through pilot stages, proving that the technology can work. We are in the midst of a capital expansion to scale that to commercial levels and have already signed a number of programs with customers to leverage that technology. But this is factored into our long-term confidence in this business segment as well in terms of its ability to grow at the 5% to 7% in the long term. But those are -- in terms of Zydis Ultra, we're talking further out before we'd start to see meaningful revenue from Zydis Ultra.

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Operator [7]

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Our next question comes from Dan Brennan with UBS.

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Daniel Gregory Brennan, UBS Investment Bank, Research Division - Senior Equity Research Analyst of Healthcare Life Sciences [8]

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Congrats on the quarter and the deal. First, just on Paragon, just -- can you give us a little flavor? It came in other than we expected this quarter, I guess, not surprising given the commentary intra-quarter and the overall market. But can you give us a little flavor for kind of what you're seeing there? And then secondarily, can you kind of clarify a little bit on the increased CapEx plans, kind of any clarification on kind of what the future revenue contribution as you build-out the capacity in Paragon? Because I know, John, you've alluded to that in your prepared remarks.

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Edited Transcript of CTLT earnings conference call or presentation 3-Feb-20 1:15pm GMT - Yahoo Finance

Recommendation and review posted by Bethany Smith

The complete range of information related to the Gene Therapies Market is obtained through various sources and this obtained the bulk of the information is arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis to generate a whole set of trade based study regarding the Gene Therapies Market.

ReportsnReports added a new report on The Gene Therapies Market report delivers the clean elaborated structure of the Report comprising each and every business related information of the market at a global level.

Download FREE PDF Sample of this Report @ http://www.reportsnreports.com/contactme=1344314

Gene Therapies: A Growing Pipeline and Approval of CAR-T Cell Therapies Reflect Increasing Commercial Appeal, Despite the Difficulties Faced by products such as Glybera and Strimvelis discusses all gene therapies, including not only those that involve genetic modification, but also RNAi, CAR-T cell and aptamer-based therapies.

In 2017 alone, the marketing approvals of Kymriah, Yescarta and Luxturna added to this growing class of products. However, due to various challenges, progress in developing this technology and achieving commercial uptake over the past few decades has been slow.

In the short term, there is a shortfall in global gene and cell therapy manufacturing capacity, and in the long-term the personalized nature of many gene and cell therapies represents a further challenge that will not be met by the currently used, centralized biopharmaceutical manufacturing model.

Additionally, the very small number of patients in orphan genetic diseases that these therapies are especially well suited for has caused commercial difficulties for drugs such as Glybera and Strimvelis, and the high pricing of these therapies in response to the low patient number presented strong reimbursement difficulties.

However, the pipeline for gene therapies is robust; there are 985 in vivo gene therapies, and a further 354 CAR-T cell therapies currently in pipeline development.

Most are at an early stage of development, with 76% at the Discovery or Preclinical stage.

There are also currently 23 gene therapy programs in Phase III development. This report provides a comprehensive view of the clinical, R&D, commercial and competitive landscape of Gene Therapy, and assesses key developments in delivery vector technology, and challenges and advances associated with the production of such vectors.

Reasons to accessthis report:

Understand the current status of the field of therapeutic gene therapies, and the relative clinical and commercial success of currently marketed products,Assess the pipeline for gene therapies split by therapy area, vector type and intervention type, and stage of development. Additionally, a granular assessment of the pipeline is provided across the four major therapy areas for gene therapy: oncology, central nervous system disorders, ophthalmology, and genetic disorders, Gain a picture of the current competitive landscape, with a detailed breakdown of companies actively involved in the gene therapy pipeline, Understand the level of involvement in the landscape on the part of big pharma companies, and the extent to how gene therapies fit into the overall portfolios of companies in this field,Understand the strategic consolidations landscape in gene therapies across the past decade.

Access this Report @ http://www.reportsnreports.com/purchasme=1344314

Scope of this report:

Why do gene therapies still occupy only a minimal market share in their respective indications?,What can be learned from the gene therapies that have already reached the market, especially from products that have faced commercial difficulties, such as Glybera and Strimvelis?,What are the prospects of recent approvals such as Luxturna, Kymriah and Strimvelis?,What are the relative advantages and disadvantages of each vector type and which hold the most promise?,What proportion of the overall gene therapy R&D pipeline is occupied by each vector type?,How will the entry of gene therapies into the market change the global manufacturing landscape?

Table of Contents for Gene Therapies Market:

1 Table of Contents 2

1.1 List of Tables 3

1.2 List of Figures 3

2 Gene Therapy Overview 5

2.1 Types of Gene Therapy 7

2.1.1 Types of Intervention 8

2.1.2 Types of Vector 13

3 Currently Approved Gene Therapies 22

3.1 Glybera (alipogene tiparvovec) 22

3.2 Kynamro (mipomersen) 23

3.3 Macugen (pegaptanib) 24

3.4 Vitravene (fomivirsen) 25

3.5 Gendicine (rAd-p53) 26

3.6 Oncorine (rAd5-H101) 26

3.7 Neovasculgen (Pl-VEGF165) 27

3.8 Exondys 51 (eteplirsen) 28

3.9 Spinraza (nusinersen) 29

3.10 Strimvelis (GSK-2696273) 30

3.11 Kymriah (tisagenlecleucel) 32

3.12 Yescarta (axicabtagene ciloleucel) 33

3.13 Imlygic (talimogene laherparepvec) 34

3.14 Zalmoxis (Allogenic T cells encoding LNGFR and HSV-TK) 36

3.15 Luxturna (voretigene neparvovec) 37

4 Gene Therapy Production Strategies 38

4.1 Production of Viral Vectors 38

4.1.1 Case Study: Challenges in the Manufacture of AAV Vectors 40

4.2 Production of Cell-based Gene Therapies 42

5 Challenges to Gene Therapy Development 44

6 Gene Therapy Pipeline and Emerging Technologies 46

6.1 Gene Therapy Pipeline by Therapy Area and Stage of Development 46

6.2 Gene Therapy Pipeline by Intervention and Vector Type 48

6.3 Pipeline for CAR-T Cell Therapies 51

6.4 Company Positioning 52

6.4.1 Companies by Therapy Area 53

6.4.2 Companies by Stage of Development 54

6.4.3 Companies by Intervention Type 54

6.4.4 Companies by Vector Type 56

6.4.5 Companies Developing CAR-T Cell Therapies by Stage 57

6.5 Early Genome Editing Technologies 57

6.5.1 Zinc Finger Nucleases 58

6.5.2 TALEN 58

6.5.3 CRISPR-Cas9 59

7 Strategic Consolidations 62

7.1 Licensing Deals 62

7.1.1 Licensing Deals by Region Value and Year 62

7.1.2 Licensing Deals by Stage of Development and Value 64

7.1.3 Licensing Deals by Intervention and Vector Type 65

7.2 Co-development deals 66

7.2.1 Co-development Deals by Region, Year and Value 66

7.2.2 Co-development Deals by Stage of Development and Value 68

7.2.3 Co-development Deals by Intervention and Vector Type 69

Access this report @ http://www.reportsnreports.com/contactme=1344314

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Original post:
New release: 2020 Gene Therapies Market Report - WhaTech Technology and Markets News

Recommendation and review posted by Bethany Smith

Exosome Diagnostics Market Report analysis including industry Overview, Country Analysis, Key Trends, Key Retail Innovations, Competitive Landscape and Sector Analysis for upcoming years.

ReportsnReports added a new report on The Exosome Diagnostics Market Technologies report delivers the clean elaborated structure of the Report comprising each and every business related information of the market at a global level. The complete range of information related to the Exosome Diagnostics Market Technologies is obtained through various sources and this obtained the bulk of the information is arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis to generate a whole set of trade based study regarding the Exosome Diagnostics Market Technologies.

Download a Free PDF Sample of Exosome Diagnostics Market Technologies Research Report at:

http://www.reportsnreports.com/contactme=1781607

Top Companies mentioned in this report are Capricor Therapeutics Inc, Evox Therapeutics Ltd, ReNeuron Group Plc, Stem Cell Medicine Ltd, Tavec Inc, Codiak Biosciences Inc, Therapeutic Solutions International Inc, ArunA Biomedical Inc, Ciloa 85.

This latest report is on Exosome Diagnostics Market Technologies which explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.

These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field.

Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy.

Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intracellular transportation of genetic material to target cells.

As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biological vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes.

Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.

Reasons to buy this Report:

Develop a comprehensive understanding of exosome technologies and their potential for use within the healthcare sector, Analyze the pipeline landscape and gain insight into the key companies investing in exosomes technologies, Identify trends in interventional and observational clinical trials relevant to exosomes.

Get this Report @ http://www.reportsnreports.com/purchasme=1781607

Scope of this Report:

What are the features of the exosome lifecycle?,How are therapeutic exosomes prepared?,How do exosome therapies in development differ in terms of stage of development, molecule type and therapy area?,Which companies are investing in exosome technologies?,How many clinical trials investigate exosomes as biomarkers, therapeutics and vectors?

Table of contents for Exosome Diagnostics Market Technologies:

1 Table of Contents 4

1.1 List of Tables 6

1.2 List of Figures 7

2 Exosomes in Healthcare 8

2.1 Overview of Exosomes 8

2.2 Drug Delivery Systems 9

2.2.1 Modified Release Drug Delivery Systems 9

2.2.2 Targeted Drug Delivery Systems 10

2.2.3 Liposomes 12

2.2.4 Viruses 14

2.2.5 Exosomes 17

2.3 The Exosome Lifecycle 18

2.4 Exosomes in Biology 18

2.5 Exosomes in Medicine 19

2.5.1 Biomarkers 19

2.5.2 Vaccines 20

2.6 Exosomes as a Therapeutic Target 20

2.7 Exosomes as Drug Delivery Vehicles 21

2.8 Therapeutic Preparation of Exosomes 21

2.8.1 Isolation and Purification 22

2.8.2 Drug Loading 22

2.8.3 Characterization 23

2.8.4 Bioengineering 23

2.8.5 Biodistribution and In Vivo Studies 23

2.8.6 Advantages of Exosome Therapies 24

2.8.7 Disadvantages of Exosome Therapies 24

2.9 Exosomes in Therapeutic Research 25

2.9.1 Exosome Gene Therapies 25

2.9.2 Exosome in Stem Cell Therapy 26

2.10 Exosomes in Oncology 27

2.10.1 Immunotherapy 27

2.10.2 Gene Therapy 28

2.10.3 Drug Delivery 29

2.10.4 Biomarkers 30

2.11 Exosomes in CNS Disease 30

2.11.1 Tackling the Blood-Brain Barrier 30

2.11.2 Exosomes in CNS Drug Delivery 31

2.11.3 Gene Therapy 32

2.12 Exosomes in Other Diseases 33

2.12.1 Cardiovascular Disease 33

2.12.2 Metabolic Disease 33

3 Assessment of Pipeline Product Innovation 36

3.1 Overview 36

3.2 Exosome Pipeline by Stage of Development and Molecule Type 36

3.3 Pipeline by Molecular Target 37

3.4 Pipeline by Therapy Area and Indication 38

3.5 Pipeline Product Profiles 38

3.5.1 AB-126 - ArunA Biomedical Inc. 38

3.5.2 ALX-029 and ALX-102 - Alxerion Biotech 39

3.5.3 Biologics for Autism - Stem Cell Medicine Ltd 39

3.5.4 Biologic for Breast Cancer - Exovita Biosciences Inc. 39

3.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis - Regenasome Pty 39

3.5.6 Biologic for Lysosomal Storage Disorder - Exerkine 39

3.5.7 Biologics for Prostate Cancer - Cells for Cells 40

3.5.8 CAP-2003 - Capricor Therapeutics Inc. 40

3.5.9 CAP-1002 - Capricor Therapeutics Inc. 41

3.5.10 CIL-15001 and CIL-15002 - Ciloa 42

3.5.11 ExoPr0 - ReNeuron Group Plc 42

3.5.12 MVAX-001 - MolecuVax Inc. 43

3.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke - Isfahan University of Medical Sciences 44

3.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer - Codiak BioSciences Inc. 44

3.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology - Evox Therapeutics Ltd 44

3.5.16 TVC-201 and TVC-300 - Tavec Inc. 45

4 Assessment of Clinical Trial Landscape 48

4.1 Interventional Clinical Trials 48

4.1.1 Clinical Trials by Therapy Type 48

4.1.2 Clinical Trials by Therapy Area 49

4.1.3 Clinical Trials by Stage of Development 50

4.1.4 Clinical Trials by Start Date and Status 50

4.2 Observational Clinical Trials 51

4.2.1 Clinical Trials by Therapy Type 51

4.2.2 Clinical Trials by Therapy Area 51

4.2.3 Clinical Trials by Stage of Development 52

4.2.4 Clinical Trials by Start Date and Status 53

4.2.5 List of All Clinical Trials 54

5 Company Analysis and Positioning 67

5.1 Company Profiles 67

5.1.1 Capricor Therapeutics Inc. 67

5.1.2 Evox Therapeutics Ltd 72

5.1.3 ReNeuron Group Plc 73

5.1.4 Stem Cell Medicine Ltd 77

5.1.5 Tavec Inc. 78

5.1.6 Codiak Biosciences Inc. 80

Original post:
Current research: 2020 Latest Report on Exosome Diagnostics Market Report Technologies, Analyze the Pipeline Landscape and Key Companies - WhaTech...

Recommendation and review posted by Bethany Smith

BOSTON--(BUSINESS WIRE)--Decibel Therapeutics, a development-stage biotechnology company developing novel therapeutics for hearing loss and balance disorders, today announced a new strategic research focus on regenerative medicine approaches for the inner ear. The company is also announcing a collaboration and option agreement that gives Decibel exclusive access to novel compounds targeting proteins in a critical regenerative pathway.

Decibels research focus on regeneration will be powered by the companys research and translation platform. The company has built one of the most sophisticated single cell genomics and bioinformatics platforms in the industry to identify and validate targets. Decibel has also developed unique insights into regulatory pathways and inner ear delivery mechanisms that together enable precise control over gene expression in the inner ear and differentiate its AAV-based gene therapy programs.

Our deep understanding of the biology of the inner ear and our advanced technological capabilities come together to create a powerful platform for regenerative medicine therapies for hearing and balance disorders, said Laurence Reid, Ph.D., acting CEO of Decibel. We see an exciting opportunity to leverage this platform to address a broad range of hearing and balance disorders that severely compromise quality of life for hundreds of millions of people around the world.

The first program in Decibels regeneration portfolio aims to restore balance function using an AAV-based gene therapy (DB-201), which utilizes a cell-specific promoter to selectively deliver a regeneration-promoting gene to target cells. In collaboration with Regeneron Pharmaceuticals, Decibel will initially evaluate DB-201 as a treatment for bilateral vestibulopathy, a debilitating condition that significantly impairs balance, mobility, and stability of vision. Ultimately, this program may have applicability in a broad range of age-related balance disorders. There are currently no approved medicines to restore balance. Decibel expects to initiate IND-enabling experiments for this program in the first half of 2020.

Decibel is also pursuing novel targets for the regeneration of critical cells in both the vestibule and cochlea of the inner ear; these targets may be addressable by gene therapy or other therapeutic modalities. As a key component of that program, Decibel today announced an exclusive worldwide option agreement with The Rockefeller University, which has discovered a novel series of small-molecule LATS inhibitors. LATS kinases are a core component of the Hippo signaling pathway, which plays a key role in regulating both tissue regeneration and the proliferation of cells in the inner ear that are crucial to hearing and balance. The agreement gives Decibel an exclusive option to license this series of compounds across all therapeutic areas.

The agreement also establishes a research collaboration between Decibel and A. James Hudspeth, M.D., Ph.D., the F.M. Kirby Professor at The Rockefeller University and the director of the F.M. Kirby Center for Sensory Neuroscience. Dr. Hudspeth is a world-renowned neuroscientist, a member of the National Academy of Sciences and the American Academy of Arts and Sciences, and a Howard Hughes Medical Institute investigator. Dr. Hudspeth has been the recipient of numerous prestigious awards, including the 2018 Kavli Prize in Neuroscience.

Rockefeller scientists are at the leading edge of discovery, and we are excited to see the work of Dr. Hudspeth move forward in partnership with Decibel, said Jeanne Farrell, Ph.D., associate vice president for technology advancement at The Rockefeller University. The ambitious pursuit of harnessing the power of regenerative medicine to create a new option for patients with hearing loss could transform how we address this unmet medical need in the future.

In parallel with its new research focus on regenerative strategies, Decibel will continue to advance key priority preclinical and clinical programs. DB-020, the companys clinical-stage candidate designed to prevent hearing damage in people receiving cisplatin chemotherapy, is in an ongoing Phase 1b trial. Decibel will also continue to progress DB-OTO, a gene therapy for the treatment of genetic congenital deafness, which is being developed in partnership with Regeneron Pharmaceuticals. The DB-OTO program aims to restore hearing to people born with profound hearing loss due to a mutation in the otoferlin gene and is expected to progress to clinical trials in 2021.

To support the new research focus, Decibel is restructuring its employee base and discontinuing some early-stage discovery programs.

About Decibel Therapeutics, Inc.Decibel Therapeutics, a development-stage biotechnology company, has established the worlds first comprehensive drug discovery, development, and translational research platform for hearing loss and balance disorders. Decibel is advancing a portfolio of discovery-stage programs aimed at restoring hearing and balance function to further our vision of a world in which the benefits and joys of hearing are available to all. Decibels lead therapeutic candidate, DB-020, is being investigated for the prevention of ototoxicity associated with cisplatin chemotherapy. For more information about Decibel Therapeutics, please visit decibeltx.com or follow @DecibelTx.

Read the original post:
Decibel Therapeutics Announces Strategic Research Focus on Regenerative Medicine for the Inner Ear - Business Wire

Recommendation and review posted by Bethany Smith

The biotech Hotbed known as Pharm Country, which includes New Jersey, Connecticut, New York Pennsylvania and Rhode Island, is an area of rapid growth and change. As a result, its no wonder that some of the biggest companies in the world call this Hotbed home.

In BioSpaces recently published 2019 Life Sciences Ideal Employer Report, more than 2,700 life sciences professionals weighed in on their Ideal Employers. BioSpace broke down the data to gain an insight into each of the BioSpace Hotbed regions and who those survey takers thought should be considered Ideal Employers. Similar to the Biotech Bay list and the top 10 list for Genetown, BioSpace takes a look at the top 10 2019 Life Sciences Ideal Employer Reports for Pharm Country.

As BioSpace notes in its Pharm Country report, not all of the companies included in this region made the 2019 Ideal Employer list, such as Janssen, Novo Nordisk and Spark Therapeutics. Some companies that did rank on the Ideal Employer List, such as Regeneron, rank higher here, most likely due to the fact its headquarters is located in Tarrytown, New York.

See more here:
Pharm Country Professionals Weigh In on Their Ideal Employers - BioSpace

Recommendation and review posted by Bethany Smith

Top research study on Global Personalized Gene Therapy Treatments For Cancer Market is an extensive compilation of innovative developments, growth opportunities and revenue analysis of top-tier Personalized Gene Therapy Treatments For Cancer Industry aspirants. The report states the growth trajectory of Global Personalized Gene Therapy Treatments For Cancer Market growth during 2020-2026. Key industry aspects like SWOT analysis, Porters five forces analysis, and market statistics are mentioned. Global Personalized Gene Therapy Treatments For Cancer Industry is expected to reach xx million USD in 2020 and will grow at a CAGR of xx% during 2020-2026.

Checkout TOC And Research Coverage With Important Details Here: https://reportscheck.biz/report/44134/global-personalized-gene-therapy-treatments-for-cancer-industry-market-research-report/#table-of-content

The prime manufacturers of Personalized Gene Therapy Treatments For Cancer Market is as follows:

GenprexGenesysCelsionAdaptimmuneAdvantageneAmgenMaxiVaxBioCancellCelgeneBlubird BIOOncoSecBellicum

The production, regional trade, investment opportunities, mergers & acquisitions and sales channels of Personalized Gene Therapy Treatments For Cancer Industry are stated. The top manufacturers, product types, applications, and market share is stated. The regional Personalized Gene Therapy Treatments For Cancer analysis covers North America, Europe, China, Japan, India, South America, Middle East, and Africa and the rest of the world.

The product types covered in the report are as follows:

Type 1Type 2Type 3Type 4Type 5

The top applications in Personalized Gene Therapy Treatments For Cancer Market are as follows:

Application 1Application 2Application 3Application 4Application 5

Global Personalized Gene Therapy Treatments For Cancer Research Report offers complete details about industry chain structure, raw materials, pricing analysis, company profiles, and product specifications. The sales analysis, value chain optimization, strategic insights on Personalized Gene Therapy Treatments For Cancer Industry, product launches and market risks are mentioned in this report. The country-level analysis of Personalized Gene Therapy Treatments For Cancer Report covers USA, Canada, Mexico, Germany, France, UK, Russia, Italy, China, Japan, India, Korea, Australia, Brazil, Argentina, Colombia, Saudi Arabia, UAE, Egypt, South Africa and rest of the world. Market scope, revenue, information on product services and gross margin status is covered in this report. The import-export scenario, demand-supply, consumer behavior, and complete details on distributors, suppliers, traders, and dealers in Personalized Gene Therapy Treatments For Cancer Market are stated.

Know More About This Report Or Request Free Sample With Custom Queries (If Any): https://reportscheck.biz/report/44134/global-personalized-gene-therapy-treatments-for-cancer-industry-market-research-report/#sample-report

Key Extracts From Table Of Content:

Section 1: Market Introduction and Overview

Section 2: Product Overview, Classification, Scope

Section 3: Competitive Personalized Gene Therapy Treatments For Cancer Market scenario based on Top Manufacturers

Section 4: Historic Study of Personalized Gene Therapy Treatments For Cancer Market Based on Region, Type, Application

Section 5: Company Profiles of Key Personalized Gene Therapy Treatments For Cancer Players, Market Share, Product Portfolio and Regional Presence

Section 6: Manufacturing Cost Analysis, Key Business Figures, Gross Margin, SWOT Analysis

Section 7: Regional Analysis, Market Status and Prospect From 2015-2026

Section 8: Market Dynamics, Marketing and Sales Channels, Distributors and Customer Analysis

Section 9: Financial Highlights of Personalized Gene Therapy Treatments For Cancer Market Including Total Revenue, Products, Services, Opportunities, and Market Risk Analysis

Section 10: Global Personalized Gene Therapy Treatments For Cancer Market Forecast Study, Marketing Channels, Cost Structures, Distributors and Consumer Study

Section 11: Region-wise Forecast Analysis of Sales, Revenue, Growth Rate Till 2026

Section 12: Research Findings, Conclusion, Data Sources, Research Methodology, and Disclaimer

A complete qualitative and competitive assessment of Personalized Gene Therapy Treatments For Cancer Market is conducted to offer valuable insights. This will enable the market aspirants in shaping their business plans and planning growth strategies. Primary and secondary research techniques like interviews, trade journals, surveys, and reputable paid database sources. A complete historical analysis from 2014-2019 and forecast analysis 2020-2026 with base year as 2019. Our competitive business landscape will help you to gain upper hand in competition.

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Global Personalized Gene Therapy Treatments For Cancer Market 2020 Reflects Accelerated Growth Driven By Rapid Urbanization And Changing Consumer...

Recommendation and review posted by Bethany Smith

Fior Marketshas the latest research report titledGlobalGene TherapyMarket Growth 2019-2024which presents the clean elaborated structure of the report comprising an in-depth evaluation of this industry and a commendable brief of its segmentation. Our team of expert researchers has obtained the complete range of information related to the globalGene Therapymarket. The information gathered from different sources has been arranged, processed, and represented by a group of specialists through the application of different methodological techniques and analytical tools such as SWOT analysis.

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The research study divides the globalGene Therapymarket by top players/brands, regions, types, and end-user. It comprehensively delivers the market portion, improvement, patterns, and expectations for the period 2019-2024. The market is observing the arrival of local vendors entering the market. Additionally, key countries expected to display significant growth prospects in the future are included in the report. Moreover, the report provides key market dynamics, the profile of key market players, and a comprehensive outline of the market environment in terms of sales and production for the forecast period 2019-2024.

Key manufacturersare included based on the company profile, sales data and product specifications, etc.:Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene

The exploration report contains the board investigation of the geographical scene of the market, which is evidently arranged into the locales

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The report contains a thorough overview of the competitive backdrop of the globalGene Therapymarket that encompasses leading firms. A concise synopsis of all the manufacturers, a product developed, and product application scopes have been included. The study calculates the sales registered by the products as well as the revenues earned over the foreseeable duration. The revenues accumulated by these applications as well as the sales projections for the projected timeframe are also included in the report. Data with respect to the market dynamics such as the potential growth opportunities, challenges have been covered in this report.

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Concluding part of the reportoffers various traders, contributors engaged in theGene Therapyindustry along with research discoveries, results, data source and postscript.

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Global Gene Therapy Market 2019 By Demand drivers Bluebird Bio, Sangamo, Spark Therapeutics - Jewish Life News

Recommendation and review posted by Bethany Smith

The UK must break away from Europes restrictive agricultural gene-editing rules, science writer Matt Ridley told the UKs House of Lords on January 30. A vocal advocate for technological innovation, Ridely argued that Britain should embrace CRISPR and other new breeding techniques, or risk falling behind enterprising nations, including the US, Canada and Argentina, that have recognized the technologys benefits.

While critics warn that gene-edited crops and animals could pose a risk to human health and the environment, Ridely countered that no data support such concerns and offered several examples of how Britain stands to gain by taking an evidence-based position on CRISPR:

There is no clearer case of a technology in which we could and should take the lead, but in which we are and will be held back if we do not break free from the EU approach. That would not be a race to the bottom but the very opposite: a race to the top.

For example, if we allowed the genome-edited blight-resistant potatoes developed at the Sainsbury Laboratory to be grown here in the UK, we would be able to greatly reduce the spraying of fungicides on potato fields, which happens up to 15 times a year, harming biodiversity and causing lots of emissions from tractors. That would be an improvement, not a regression, in environmental terms.

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Video: House of Lords member and science writer Matt Ridely urges UK to 'break free' of Europe's restrictive CRISPR crop rules - Genetic Literacy...

Recommendation and review posted by Bethany Smith

SEOUL, South Korea, Feb. 3, 2020 /PRNewswire/ --Scientists at Dongguk University successfully treated Alzheimer's disease in mice using the gene editing tool CRISPR-Cas9. They used the tool to edit out the Alzheimer's causing gene in the mice, thereby improving their memory and other cognitive functions. While this is only a first step and much research remains to be done, these promising results put gene therapy on the map of possible treatments for Alzheimer's.

As Alzheimer's disease continues to elude the efforts of modern medicine, scientists turn to gene editing technology to treat, or 'manage' it. Alzheimer's is commonly associated with the BACE 1 gene, which drives the production of amyloid-proteins in the brain. In the brains of Alzheimer's patients, this protein accumulates on the outsides of neurons or nerve cells as "plaque," and is thought to be one of the main causes of the disease. Using "DNA scissors" called CRISPR-Cas9, which can cut out a specified part of a DNA sequence, Dr. Jongpil Kim and his colleagues from Korea (Dongguk University) and the USA attempted to edit out the BACE 1 gene in the neurons of the adult mouse brain.

Theirs is the first in vivo attempt (using cells inside living mice) to test the effectiveness of CRISPR-Cas9 in stopping Alzheimer's progression. Their ultimate ambitionlike that of many others in the fieldis to develop treatments for different forms of dementia, which would dramatically improve patients' quality of life. "We aimed to see whether CRISPR-Cas9, one of the latest developments in biotechnology, can open up a new direction for treating dementia, which is, at present, considered an incurable disease," Dr. Kim says.

Their results indeed look promising. Within 8 to 12 weeks of treatment with the CRISPR-Cas9 technology, the plaque surrounding affected neurons in the mice dramatically decreased and their cognitive functions improved substantially. The mice also showed better associative learning and spatial working memory. Further, there was no evidence of increasing mutations in other, non-targeted parts of the genome, suggesting minimal side-effects.

All this indicates the huge potential of gene editing technologies to treat neurodegenerative diseases: by removing faulty genes at their source, disease progression can be halted, or even reversed, in just a few weeks.

The authors advise caution, however, as gene editing cannot be undone. In Dr. Kim's words, one must ensure that "no detrimental, and potentially very rare, genomic alterations are caused". Thus, this technique requires much more research before it can be applied to human subjects.

Nonetheless, as another remark by Dr. Kim goes, "this study shows how CRISPR-Cas9 can be applied to the treatment of neurodegenerative diseases as well."

Reference

Author:

Jongpil Kim

Title of original paper:

In vivo neuronal gene editing via CRISPRCas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease

Journal:

Nature Neuroscience

DOI:

10.1038/s41593-019-0352-0

Affiliations:

Departments of Chemistry and Biomedical Engineering, Dongguk University

Media contact:Jongpil Kim 233458@email4pr.com+82-10-4013-3685

SOURCE Dongguk University

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DNA "Scissors" Could Cut Out the Alzheimer's Causing Gene in Mice - PRNewswire

Recommendation and review posted by Bethany Smith

ClinGen is funded by the National Human Genome Research Institute (NHGRI), which is itself part of the National Institutes of Health. Erin Ramos, PhD, a project scientist for ClinGen and program director for the division of genomic medicine at NHGRI, said in a release that NHGRI developed ClinGen as a way to standardize guidelines for genetic testing. A panel of experts, including researchers, clinicians and genetic counselors comb through scientific evidence from research papers to identify gene-disease relationships as either definitive, strong, moderate, limited, disputed or refuted.

The experts reported that three genesKCNQ1, KCNH2 and SCN5Awere indeed backed by enough evidence to be categorized as definitive genetic triggers for long QT syndrome. They classified four other genes as either strong or definitive for causing atypical forms of long QT syndrome, but the panel didnt find enough evidence to support the remaining 10 genes.

Those 10 genes were all placed in either the limited or disputed category for their link to long QT syndrome, and for that reason the authors said they dont recommend using them as markers in routine clinical tests.

Our study highlights the need to take a step back and to critically evaluate the level of evidence for all reported gene-disease associations, especially when applying genetic testing for diagnostic purposes in our patients, Gollob said in a statement. Testing genes with insufficient evidence to support disease causation only creates a risk of inappropriately interpreting the genetic information and leading to patient harm.

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Experts dispute 9 of 17 genes once linked to long QT syndrome - Cardiovascular Business

Recommendation and review posted by Bethany Smith

KOKOMO In October 2011, Amber Broman remembers sitting in the doctors office bawling, screaming and crying out.

Minutes earlier, she had learned that the baby girl she was carrying would likely never have the chance to talk or walk, ride a bike or drive a car or be walked down the aisle on her wedding day.

We had an amniocentesis and had noticed some abnormalities in the ultrasound, Broman noted. So we saw a specialist in Indianapolis and did an amnio there too. On the ultrasound, they initially saw multiple defects within her heart, and they didnt think her esophagus was attached to her stomach. And thats when they told me what it was and that it was in every cell of her body.

The condition was Trisomy 18.

It was five weeks before the Kokomo woman was to deliver, and all she remembers hearing that day were the words incompatible with life.

According to the National Institutes of Health, Trisomy 18 is a chromosomal genetic disorder that includes a combination of birth defects that impact nearly every organ in the body. People born with the disorder, also known as Edwards syndrome, have three copies of the 18th chromosome, and most babies with Trisomy 18 die within the first year of life.

She only lived two days, Broman said, referring to her daughter she named Khloe Nicole. We chose comfort care for her because we did not necessarily want to go through having her on machines and then making those decisions. We chose to just let life take its course, and we had almost 48 full hours with her.

Broman is not alone.

The CDC describes a birth defect as a structural change present at birth that can affect any or all parts of the body. They vary from mild to severe and depending on the type of defect, some infants can go on to live healthy lives.

In the United States, a baby is born with a birth defect every four-and-a-half minutes, according to the Indiana Birth Defects and Problems Registry, which equals roughly 120,000 babies per year.

In the Hoosier state, birth defects are the second leading cause of death for infants, with over 2,500 Indiana babies born each year with some type of congenital abnormality, such as a cardiovascular, chromosomal, central nervous system or musculoskeletal defect.

In 2018 alone, the Indiana State Department of Health concluded that birth defects contributed to 6.8 deaths per 1,000 births, a slight dip from 2017.

The infant mortality rate in Indiana is the lowest its been in six years, the ISDH noted, but the rate is still one of the highest in the nation.

In recent months, Gov. Eric Holcomb has taken the infant mortality plight to the public stage, making it one of the top priorities in his administration to have the lowest infant mortality rate in the Midwest by 2024.

The state has already begun the process of achieving that goal, rolling out programs and committees with the sole focus of lowering the infant mortality rate.

One of those programs, OB Navigator, was signed into law in 2019 and works in collaboration with the ISDH, the Indiana Family and Social Services Administration and the Indiana Department of Child Services.

OB Navigator was established by Indiana House Enrolled Act 1007 and is set in place to build a network of services and support throughout Indiana to wrap our arms around moms and babies to create healthier outcomes for both, its website states.

The program works as a connection tool between women who are early in their pregnancies and individuals who can provide personal guidance for those women through the rest of those pregnancies and at least the first six months of life.

Some of that guidance includes a Moms Helpline, which is a contact center that connects pregnant women throughout Indiana to available resources and a network of prenatal and child health care services, the programs website cites.

In a recent press release on the subject, State Health Commissioner Kristina Box noted that OB Navigator will be available to women on Medicaid in the states highest-risk counties, and organizers of the program anticipate enacting it in 20 counties by the end of 2020.

Visit the ISDHs website at in.gov/isdh/ for more information about the program.

While doctors do say that many birth defects cant necessarily be fully prevented, there are ways to lower the risk factor.

We usually dont start doing testing until about the second trimester of pregnancy, said Dr. Andrew Barlow, OBGYN at Trinity Health Network. We start offering blood tests and ultrasounds at that time to determine birth defects. However, there are women that can undergo screening beforehand if theyre at higher risks.

Women that fall into those categories are those over 35 or women with certain medical conditions, Barlow noted.

Because fetal growth, like organ development, takes place in the first few weeks of pregnancy, Barlow said its imperative to see your doctor on a regular basis if youre planning on becoming pregnant.

Review your medical history and make sure youre healthy enough to have children too, Barlow added. And make sure that youre not taking any medication that can contribute to birth defects. Also obviously dont abuse alcohol or drugs during pregnancy either.

Its also important to take a prenatal vitamin with the right amount of folic acid [400 mg], Barlow added, which having a deficiency of can lead to brain and spine defects like spina bifida or anencephaly. Spina bifida is a birth defect in which a developing babys spinal cord fails to develop properly. Anencephaly is the absence of a major portion of the brain, skull, and scalp that occurs during the development of the embryo.

Dr. Sheila Hockman, OBGYN at Ascension Medical Group and part of a statewide Maternal Mortality Review Committee enacted to address the issue, agreed with Barlow, while also stressing that, such as in cases like young Khloes, there is really nothing that mothers can do to prevent such a defect.

However, areas like genetic testing can still be a giant help, even if a defect is already in place, she noted.

You can do genetic testing early in the pregnancy, Hockman said. And a lot of people, when we ask them if they want to do genetic testing, theyll say no because theyre going to deliver the baby regardless.

But for us, its not a matter of keeping the pregnancy or terminating it, she continued. Its the more well-prepared we are during the pregnancy, it enables us to care possibly in a different fashion. Do they need to see a maternal fetal medical specialist or can this pregnancy be monitored like a regular uncomplicated pregnancy? Its that sort of thing.

That can sometimes mean the difference between life and death, Hockman added.

The big point I would like to get across is that there are so many of these birth defects that are preventable, especially when you look at things like maternal obesity, she said. Its a national issue, but we can do something about it, and it often just takes a healthier lifestyle.

Continue reading here:
Birth defects second leading cause of death for infants in Indiana - The Herald Bulletin

Recommendation and review posted by Bethany Smith

Dell Medical School at The University of Texas Austin held its second public interest meeting on conducting a memory loss cohort study with Georgetown residents Jan. 31.

Survey results conducted between October and December to gauge public interest in participating in a long-term research study were presented during the meeting.

Results showed that of 279 respondents, 85% were interested in memory and learning activities associated with Alzheimers and dementia. The survey allowed takers to select multiple interests and also found that 72% of respondents were interested in exercise and diet and nutrition as it relates to memory loss.

In addition, 95% of respondents were willing to be assessed in cognitive testing and giving blood samples. The multiselect survey also found that 84% were willing to participate in genetic testing, and 78% were willing to participate in neuroimaging studies.

Alyssa Aguirre, manager of the Cognitive Disorders Center at the Mulva Clinic for the Neurosciences at Dell Medical School, said that the results make sense, as oftentimes, people want to be proactive and learn how they can prevent the disease from occurring.

The study, which would be conducted and partially funded through the Mulva Clinic for the Neurosciences at Dell Medical School, is in its early stages and would still need to be designed before it begins, Aguirre said.

Aguirre said she hopes that resident input will help the school to have studies designed around resident interests.

Other questions found that 94% of people were willing to volunteer as a participant of the study, and 89% of people were willing to be assessed every six months.

For more on the study, click here.

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Georgetown residents show high interest in participating in Alzheimer's and dementia study - Community Impact Newspaper

Recommendation and review posted by Bethany Smith

The growth of the cannabis testing market is primarily attributed to the European government funding cannabis education for doctors, and the increasing demand for research activities for cannabis quality.

A new report, entitled Europe Cannabis Testing Market to 2025 Regional Analysis and Forecasts by Type; Services; End User and Country, has outlined the growth of the European testing market up until 2025, and highlights that cannabis used as a street drug and trouble with CBD oil are likely to pose a negative impact on the market growth.

On the use of cannabis in clinical practice, to instruct local healthcare providers, various cannabis experts were invited to Macedonia, in 2016. The programme was held in Skopje and was supported and funded by the Macedonian Ministry of Health.

Cannabis provides the unique opportunities in Macedonia, where the medical system is in a stage of evolution and modernisation.

Local medical specialists are now able to prescribe cannabis in the country. Considering this, Macedonian cannabis promoters worked with local governments and created the medical cannabis training programme in an effort to support doctors and their patients.

Currently, the impetus of the Macedonian medical cannabis movement is moving quickly, and the public opinion on cannabis has also changed dramatically in the region.

The medical marijuana resolution in the region urges the Commission and member states to address regulatory, financial and cultural barriers which burden scientific research and invite them to fund research. Thus, due to the above advancements, the cannabis testing market is expected to grow at a rapid pace during the forecast period.

In 2017, the product segment held a largest market share of 73.7% of the cannabis testing market, by type. This segment is also expected to dominate the market in 2025 owing a diverse portfolio of cannabis testing products to the healthcare industry for innovations in the technologies.

Moreover, the same segment is also expected to witness the highest CAGR in the market accounting to 11.1% in 2018 to 2015 owing to increasing analytical services and medicinal cannabis services in testing cannabis.

The Europe cannabis testing market, based on services was segmented into potency testing, microbial analysis, residual solvent screening, heavy metal testing, pesticide screening, terpene profiling, and genetic testing. The potency testing segment is anticipated to grow at a CAGR of 11.8% during the forecast period.

The testing laboratory segment held a largest market share of 49.2% of the cannabis testing market, by end user. This segment is also expected to dominate the market in 2025 owing to increasing number of diagnostic testing performed in the reference labs for the numerous diseases.

Some of the major primary and secondary sources for cannabis testing included in the report are Federal Commission for the Protection against Sanitary Risk (COFEPRIS), Defense Advanced Research Projects Agency (DARPA), German Research Foundation (DFG), European Research Area (ERA), Food & Drug Administration (FDA), Indian Institutes of Technology (IITs), Japan Society for the Promotion of Science (JSPS), Laboratory Information Management System (LIMS), Medical Marijuana Regulation and Safety Act (MMRSA), Non-Governmental Organization (NGO), National Science Foundation (NSF) and others.

Continued here:
European cannabis testing market expected to reach $807.9m by 2025 - Health Europa

Recommendation and review posted by Bethany Smith

A leading research firm, Zion Market Research added a latest industry report on "Global Male Hypogonadism Market" consisting of 110+ pages during the forecast period and Male Hypogonadism Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Male Hypogonadism Market.

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The Male Hypogonadism Market report mainly includes the major company profiles with their annual sales & revenue, business strategies, company major products, profits, industry growth parameters, industry contribution on global and regional level.This report covers the global Male Hypogonadism Market performance in terms of value and volume contribution. This section also includes major company analysis of key trends, drivers, restraints, challenges, and opportunities, which are influencing the global Male Hypogonadism Market. Impact analysis of key growth drivers and restraints, based on the weighted average model, is included in this report to better equip clients with crystal clear decision-making insights.

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Zion Market Research is an obligated company. We create futuristic, cutting edge, informative reports ranging from industry reports, company reports to country reports. We provide our clients not only with market statistics unveiled by avowed private publishers and public organizations but also with vogue and newest industry reports along with pre-eminent and niche company profiles. Our database of market research reports comprises a wide variety of reports from cardinal industries. Our database is been updated constantly in order to fulfill our clients with prompt and direct online access to our database. Keeping in mind the clients needs, we have included expert insights on global industries, products, and market trends in this database. Last but not the least, we make it our duty to ensure the success of clients connected to usafter allif you do well, a little of the light shines on us.

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Global Male Hypogonadism Market 2019 Comprehensive Study with On-Going Trends, Majors Players and Forecast 2025 - Global Newspaper

Recommendation and review posted by Bethany Smith

SAN ANTONIO, Feb. 3, 2020 /PRNewswire/ -- SASpine is now offering cutting edge Stem Cell Treatments to patients. For the past several years Dr. Steven Cyr, Mayo Clinic Trained Spine Surgeon, has been researching the benefits of stem cells in the treatment of multiple medical conditions including spinal disorders, specifically, conditions which involve spinal cord injury, degenerative disc disease, herniated discs, and as a supplement to enhance the success of Spinal fusions when treating instability, deformity, and fractures of the spine.

Steven J. Cyr, M.D., is a spine surgeon who has gained a reputation for surgical excellence in Texas, throughout the nation, and abroad.

Dr. Steven Cyr has been treating patients using growth factors and stem cells contained in amniotic tissue and bone marrow aspirate to provide a potential for improved success with fusion procedures, when treating herniated discs, and for arthritic or damaged joints, with remarkable success. "The goal of any medical intervention is to yield improved outcomes with the ideal result of returning a patient to normal function, when possible," states Dr Cyr. He went on to elaborate that there are times when only a structural solution can solve problems related to spinal disorders, but even in that scenario, the use of stem cells or growth factors derived from stem cell products can possibly improve the success of surgical procedures. "I have patients previously unable to jog or run return to normal function and athletic ability after injections of growth factors and stem cell products into the knee joints, hip joints, and shoulder joints," he said. "This includes high-level athletes, professional dancers, and the average weekend warrior."

There may be promise in treating patients with spinal cord injury as well. SASpine CEO, LeAnn Cyr, states, "There are reports of patients gaining significant neurological improvement after being treated with stem cells." Dr Cyr continues, "Most patients with spinal cord injuries resulting from trauma also have mechanical pressure on the nerves that result either from bone fragments or disc material compressing the spinal cord that needs to be removed along with surgical stabilization of the spinal bones. There's significant potential that stem cells bring to the equation when treating these types of patients, and I am excited about the potential that these products offer to the host of treatments to address spinal conditions and arthritic joints."

For more information about SASpine's Stem Cell Treatment Program, visit http://www.saspine.com or call (210) 487-7463 in San Antonio or (832) 919-7990 in Houston.

Related Linkswww.facebook.com/saspinewww.instagram.com/surgical.associates.in.spine

If you've been living with back pain, you're not alone. Here at SASpine, we have experienced spine specialists who are committed to improving your quality of life. (PRNewsfoto/SASpine)

View original content to download multimedia:http://www.prnewswire.com/news-releases/saspine-to-offer-stem-cell-therapy-300997355.html

SOURCE SASpine

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SASpine to offer Stem Cell Therapy - Yahoo Finance

Recommendation and review posted by Bethany Smith

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

By Denise Ryan

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to thecord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

dryan@postmedia.com

Originally posted here:
Chinese New Year babys B.C. family gives gift of life in cord - The Province

Recommendation and review posted by Bethany Smith